The Newsletter of the American Society of Gene Therapy
ASGT NEWS
FALL 2002
ASGT 5TH ANNUAL MEETING:
USE
OF STEM CELLS IN
by David Bodine, PhD
IN THIS ISSUE
GENE THERAPY
A central theme of the 2002 Annual Meeting of
the American Society of Gene Therapy was the
biology of stem cells and the use of stem cells
in gene therapy. The combination of the ability
of RNA virus vectors to integrate into the
genome of target cells and the ability of stem
cells to engraft, proliferate and differentiate
when transplanted into recipients makes stem
cells an attractive target for gene therapy
approaches. Because it is now clear that many
adult tissues contain stem cells, it is possible to
correct inherited or acquired genetic defects in
an individual’s stem cells, which would allow
regeneration of that organ with minimal
immune complications. The education program
of the ASGT Annual Meeting had several
sessions devoted to preparing new investigators
and people not actively working in the stem cell
gene therapy field so that they could fully
appreciate the scientific sessions, Dr. Margaret
Goodell of Baylor organized a popular
education session on the “ABCs” of stem cell
biology. Drs. Jane Lebkowski, Jan Nolta, and
Goodell presented the fundamentals of
embryonic stem cells, the use of immune
deficient animals for assaying human stem cells
and stem cell plasticity respectively. Another
education session organized by Dr. Robert
Hawley featured Drs. Christopher Baum,
Richard Morgan and Hawley presenting the
“ABCs” of RNA virus vectors. Due to
overwhelming demand the RNA virus session
was given twice.
Dr. Irving Weissman of Stanford University
opened the scientific program by giving the
George Stamatoyannopoulos lecture entitled
“Biology and Transplantation of Stem and
Progenitor Cells”. In an informative and
provocative presentation, Dr. Weissman
described the enrichment of mouse and human
hematopoietic stem cells and demonstrated
how this procedure could be used to study the
basic properties of these cells. Dr. Weissman
discussed how stem cells can be induced to self
renew without differentiation by specific signals
from the environment, the regulation of stem
cell number by apoptosis, the role of stem cells
in leukemia and the changes in the stem cell
phenotype and gene expression that accompany
differentiation. The discussion then turned to
the apparent ability of hematopoietic stem cells
to give rise to cells of solid organs such as the
The George Stamatoyannopoulos lecture featured Dr. Irving
Weissman presenting “Transplantation of Stem and Progenitor Cells”.
liver. Dr. Weissman preached caution in evaluating
stem cell plasticity data and set down strict
criteria for the use of the term stem cell plasticity.
Dr. Weissman concluded his lecture by describing
the findings he and others had collected about
human cloning for the National Academy of
Sciences. Weissman urged the use of embryos to
derive novel human embryonic stem cells and
described several important scientific problems
that could be addressed by the use of this
important new technology.
The stem cell theme was continued in
Workshops, Scientific Symposia and oral and
poster presentations. Three different Scientific
Symposia had stem cells as their primary focus.
Dr. John Dick described new findings showing that
human short term repopulating stem cells could
be separated from long term repopulating
hematopoietic stem cells. Dr. Markus Grompe
continued the discussion of hematopoietic stem
cells and their apparent ability to differentiate into
liver cells. Drs. John Wolfe, Howard Federoff and
Evan Snyder described the isolation,
characterization and transplantation of neuronal
stem cells and documented their ability to engraft
and differentiate into neuronal cells. Additional
findings about neuronal stem cells were reported
in an oral presentation from Dr. Kathryn Crossin’s
laboratory. A special symposium was presented in
conjunction with the International Society of Cell
Therapy. A series of talks describing different
types of stem cells and their isolation and
transplantation was highlighted by a presentation
by Dr. George Daley who demonstrated that
mouse embryonic stem cells could engraft into
the bone marrow of irradiated mice and give rise
to functional hematopoietic cells.
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ASGT 5TH ANNUAL
MEETING HIGHLIGHTS
6
MOLECULAR THERAPY
UPDATE
6
2003: ABSTRACT DEADLINE
7
ASGT OFFICERS AND
DIRECTORS
The problems associated with the introduction
of new genetic material into stem cells were
explored in a Scientific Symposium and
Workshop. Dr. Peter Quesenberry described
factors effecting the regulation of the cell cycle
in stem cells, as did an excellent oral
presentation from Dr. Nolta’s lab. Drs. Brian
Sorrentino and C. Anthony Blau described
methods for selecting or amplifying transduced
stem cells in vivo after transplantation. Dr.
Harry Malech’s lab presented similar data in an
oral session and Dr. Malech also described new
methods to culture and transduce human stem
cells for clinical use in a Scientific Symposium.
Drs. Hans-Peter Kiem and Luigi Naldini
reported improved transduction of monkey
and human hematopoietic stem cells with
RD114 pseudotyped retrovirus and lentivirus
vectors respectively. A workshop organized by
the National Heart, Lung, and Blood Institute
focused on designing gene transfer vectors for
the eventual treatment of Sickle Cell Disease
by Gene Therapy. Talks given by 10 leaders in
this field covered novel gene transfer vectors,
new ways to express genes in red blood cells,
and the latest research in stem cell selection
and amplification.
Finally, perhaps inspired by Dr. Catherine
Verfaille’s presentation on mesenchymal stem
cells at the 2001 meeting, no less than 20 oral
and poster presentations focused specifically
on these highly plastic cells. All told from a
total of almost 1,400 abstracts submitted for
the meeting, more than 300 dealt with either
the basic biology of stem cells or the
modification of stem cells in preclinical models.
ASGT, 611 EAST WELLS STREET, MILWAUKEE, WI 53202 • PHONE (414) 278-1341 • FAX: (414) 276-3349 • WEB SITE: WWW.ASGT.ORG
AMERICAN SOCIETY OF GENE THERAPY
ANNUAL MEETING HIGHLIGHTS
ASGT 5TH ANNUAL MEETING:
NON-VIRAL SUMMARY
by Richard Heller, PhD
“α - galactosidase deficiency in Fabry mice. Dr.
Yew showed how this approach could correct
this deficiency using an intravenous injection of
the vector. Dr. Carol Miao shared results on
using non-viral vectors for hemophilia B. This
approach was used to deliver a plasmid encoding
hFIX to the liver of mice. In addition to using
synthetic agents to enhance delivery, Dr. Miao
showed that enhanced transfection could be
obtained using ultrasound stimulation. Dr. James
Hecker discussed clinical applications when
short-term expression is preferable. Most
research is focused on obtaining long-term, high
expression, Dr. Hecker pointed out times when it
is more important to have short-term
expression. A presentation by Dr. Michael
Coleman discussed using non-viral gene therapy
for ischemic heart disease.
The 2002 ASGT had two excellent
education sessions for non-viral vectors.
Dr. Seng H. Cheng organized a session,
which covered important considerations
for formulation development. In this
session, Dr. Leaf Huang discussed how one
should approach systemic delivery of nonviral vectors. This is a critical area for the
future applicability of gene transfer and Dr.
Huang gave a fascinating overview of this
area. Dr. Louis Smith, covered delivery to
the muscle, a popular target for plasmid
DNA delivery. The presentation included
important points to be considered when
using synthetic vectors. Dr. Cheng also
gave an excellent presentation covering
the critical points of delivering non-viral
vectors to the lung. A second educational
session was organized by Dr. Jeffrey
Bonadio to discuss potential applications
of non-viral vectors. These vectors are
believed to have potential for DNA
vaccines, cancer therapy, tissue engineering,
and angiogenesis as well as others. Two
excellent presentations looked at potential
clinical applications. Dr. Pamela Gehron
Robey discussed the potential of a nonviral approach for targeting skeletal stem
cells and for tissue regeneration. Dr. Kristi
Anseth gave an overview of how
biomaterials will play an important role.
The sessions were both well organized and
both were presented twice to give
everyone ample opportunity to attend.
Improvements in the development of non-viral
vectors were covered in a second workshop.
Drs. Robert Debs and Kevin Rice discussed the
use of vehicles and/or carrier molecules. This
theme was extended by Dr. David Putnam in a
discussion on how phagosomolytic
microparticles could be used to deliver DNA
vaccines. Dr. Putnam showed how this was a
more effective way to deliver to antigen
presenting cells. Dr. Ruxandra Draghia-Akli, who
showed two different ways to regulate
expression following successful delivery of
plasmid DNA encoding growth hormone
releasing hormone, discussed the use of
regulated plasmids.
Potential clinical applications were
discussed in a Workshop in which four
different areas were covered. Dr. Nelson
Yew shared results from his laboratory in
using synthetic vectors for correcting an
2
Recent advances in non-viral gene transfer were
the focus of a Scientific Symposium. Dr. Jean-Paul
Behr discussed efficient ways to target cells using
nanometric DNA particles. Dr. Behr showed
how condensed particles could efficiently
transfect several different cell lines. Discussion
also covered how these particles can be coated
with PEG-folate residues to allow for extended
circulation times. Dr. Richard Heller discussed
the use of in vivo electroporation to enhance the
delivery of plasmid DNA. Dr. Heller
demonstrated the versatility of this approach for
delivering to several tissue types and for varying
expression with respect to levels and duration of
expression. The use of this delivery approach
was demonstrated showing both a therapeutic
and prophylactic effect when treating established
murine melanomas. Dr. Grayson Lipford gave an
excellent overview of CpG-DNA signaling and
the interaction with toll-like receptor 9. Dr.
Ernst Wagner presented research in the area of
tumor targeting using DNA polyplexes. Dr.
Wagner illustrated how these complexes can be
targeted to distant tumor sites following
systemic administration. In one study presented,
tumor-targeted TNF A - gene delivery induced an
anti-tumor response.
A
Gene transfer using non-viral vectors is a
rapidly growing area that has shown great
potential. The advantages of this approach
are that the vectors are generally nontoxic, have reduced immunogenicity, low
toxicity and are easier and less expensive
to prepare. These vectors are now being
used as delivery vehicles for many different
applications. The non-viral vector sessions
were extremely popular at the 2002
Annual Meeting of the American Society of
Gene Therapy as evidenced by most
sessions being filled to capacity. The 2002
meeting was an outstanding venue for
hearing the latest research in this field,
whether it was in the area of enhanced
delivery and expression or the use of nonviral vectors for therapeutic applications.
At the 2002 Annual Meeting, there were several
excellent oral and poster presentations that
covered improving the efficiency of non-viral
vectors as well as demonstrations of how this
delivery approach could be used in therapeutic
applications. Three relatively new delivery
approaches dominated these presentations,
electroporation, hydrodamics and ultra-sound.
All three of these methods have been shown to
enhance expression of plasmid DNA in a variety
of tissues. It was also interesting to note the
diversity of the therapeutic applications being
studied using non-viral vectors. The applications
of non-viral vectors discussed at this meeting
included cancer therapy, chemokine
immunotherapy, musculo-skeletal and diabetes.
Don’t forget to order your
copy of the Clinical Gene
Transfer Comprehensive
Review Course audiotapes.
There are a limited number
of sets available. The cost for
the set of ten audiotapes and
course syllabus is $53, which
includes shipping and handling.
There is an additional
shipping cost for those
ordering outside the U.S.
Feedback about the tapes has
been positive. For more
information on the tapes or
to order the tapes, please
visit the ASGT website.
WWW.ASGT.ORG
AMERICAN SOCIETY OF GENE THERAPY
ASGT 5TH ANNUAL MEETING:
CANCER GENE THERAPY HIGHLIGHTS
by Stephen Russell, MD, PhD
With 10 poster sessions, seven oral abstract
sessions, and two scientific symposia devoted
entirely or predominantly to the topic of cancer,
ASGT provided an unmissable forum for cancer
gene therapy researchers. It was clear at the
meeting that the field is brimming with novelty
and innovation and that steady progress
continues in the development of traditional
cytoreductive and immunotherapeutic
approaches.
Accurate delivery and gene expression in tumor
cells remain the keys to successful cytoreductive
gene therapy for cancer. Several ingenious vector
targeting strategies were reported at the meeting,
most notably the successful reprogramming of
adenovirus entry by genetic ablation of its natural
tropism combined with the display of polypeptide
targeting ligands incorporated into the virus coat
by Drs. Thomas Wickham and Susan Stevenson.
In this and other respects (see below),
adenovirus appears to be emerging as the
“leading light” for other vector systems, and the
early in vivo data using retargeted adenoviruses
are looking very promising.
As to optimizing the genetic payloads for
cytoreductive vectors, several new and improved
proapoptotic and suicide genes were presented.
There were also a larger number of
presentations on the use of replicationcompetent oncolytic adenovirus and herpes virus
vectors for cytoreductive gene therapy of cancer.
Drs. André Lieber, William Wold, and David
Curiel reported conditionally replicating
adenoviruses with tighter tissue specifically or
greater oncolytic potency than the well-known
prototype E1B-deleted adenovirus Onyx O15
and clinical translaton of the best of these agents
is eagerly awaited. Promising new oncolytic
agents based on measles virus and vesicular
stomatitis virus were also presented by Drs. Eva
Galanis, Glen Barber.
As an alternative to traditional (viral or non-viral)
gene therapy vectors, stem cells are being
exploited as vehicles to carry therapeutic genes
into inaccessible tumors. Genetically modified
bone marrow stem cells were shown to colonize
tumor neovessels by Dr. Luigi Naldini, and
migratory neural progenitor cells were used as
Trojan horses to migrate into the parenchyma of
intracranial gliomas and produce therapeutic
retroviral vectors in situ in the tumor.
Noninvasive imaging of gene expression
represents an enormous advance for
cytoreductive gene therapy research and was the
subject of several presentations at the meeting.
GFP and luciferase reporter genes were
exploited for noninvasive optical imaging of
intratumoral gene expression by Dr. Yoshinaga
Saeki while thymidine kinase and the
somatostatin receptor were used as reporter
genes for PET imaging by Drs. Kurt Zinn and
Andreas Jacobs. Dr. Kah-Whye Peng showed
reporter genes encoding nonimmunogenic
soluble marker peptides were also used for
noninvasive monitoring of the expression kinetics
of an oncolytic measles virus.
Immunogene therapy of cancer was also very
well represented at the meeting. This field of
research continues to grow and mature with ever
better understanding of how different tumor
cell killing scenarios impact the vigor of the
host immune response. One notable advance
was the demonstration that cell-to-cell fusion
leading to syncytial cell death is accompanied
by a particularly robust immune response
attributed in part to the release of small
subcellular particles dubbed “syncytiosomes”
which efficiently transfer tumor antigens into
dendritic cells by Dr. Richard Vile. In addition
to these mechanistic studies, Dr. Estuardo
Aguilar-Cordova demonstrated that the
spectrum of co-stimulatory molecules and
cytokines available for immunogene therapy
of cancer is expanding, and that early clinical
trial data is looking promising.
In addition to developments in cytoreductive
and immunogene therapy for cancer, there
were exciting developments in the use of
gene therapy for symptom control in cancer
patients. Examples included the use of herpes
virus vectors coding for proenkephalin for
the control of chronic cancer pain presented
by Dr. David Fink, and the use of vectors
coding for manganese superoxide dismutase
for the control of radiation-associated
mucositis presented by Dr. David Blumberg.
Overall, the vigor of cancer gene therapy
research was very strongly in evidence at this
year’s meeting, and it is difficult to envisage
anything other than continued relentless
progress and innovation by the time we are
ready for next year’s Annual Meeting.
ASGT 5TH ANNUAL MEETING:
CARDIOVASCULAR GENE THERAPY SUMMARY
by Lawrence Chan, MD
The 2002 Annual Meeting of the American
Society of Gene Therapy had many highlights
among the poster, oral, and worshop
presentations on Cardiovascular Gene Therapy. A
major target organ for gene therapy is the injured
or failing myocardium. Transgenes used for
treating myocardial ischemia-reperfusion injury
included a dominant negative IKKß gene by Dr.
Anthony Rosenzweig’s group and the heme
oxygenase-1 gene by Dr. Victor Dzau’s group
(both by intramyocardial delivery), and the
epicardial delivery of Hypoxia Inducible Factor
(HIF-1α) gene in a porcine model of chronic
myocardial ischemia by Dr. Amanda Heinl-Green
and colleagues. There were several presentations
on the delivery of VEGF, alone or in combination
with other factors, in a variety of animal models,
including myocardial ischemia and peripheral
vascular ischemia models. Dr. Jonathan Bromberg
and coworkers delivered the vIL-10 gene to
transplanted hearts that led to prolonged
allograft survival, an effect that appeared to be
mediated by the inhibition of multiple
proinflammatory chemokine and chemokine
receptors.
Many presentations explored the use of
specialized gene-transfer vectors, and others
were involved in the production of improved
versions of existing vectors. The most commonly
used vector systems were adenovirus (Ad) and
adeno-associated virus (AAV). Dr. Kazuhiro Oka
and colleagues used a helper-dependent (HD-) Ad
(devoid of all viral protein coding genes) to
deliver the apolipoprotein (apo) A-I gene to the
liver of LDL receptor-deficient mice. They
observed sustained high-level expression of
apoA-I, partial inhibition of atherosclerosis
progression and remodeling of
atheromatous plagues to a stable
phenotype. Dr. Phillip Factor and colleagues.
found that first and second generation Ads,
but not HD-Ad, increased airway reactivity
following intra-tracheal delivery in mice.
Delivery of a ß2-adrenergic receptor was
found to attenuate methacholine-induced
airway reactivity. Therefore, gene transfer
to the bronchial epithelium by HD-Ads
appears to have significantly attenuated
toxicity, an observation that is reminiscent
of the greatly improved safety profile of
HD-Ads when they were used to deliver
transgenes to the liver.
continued on page 4
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AMERICAN SOCIETY OF GENE THERAPY
CARDIOVASCULAR GENE THERAPY
continued from page 3
An important target tissue in cardiovascular gene therapy is the
vascular endothelium. The role of the vascular endothelium in gene
delivery was explored in a workshop. Dr. Philip Leopold noted that
release of inflammatory cytokines from damaged EC produced
changes in gene expression that may affect efficiency of Ad
transduction. He observed an 80% decrease in cell surface
coxsackie-adenovirus receptor (CAR) expression within 48 hours of
treatment of human umbilical vein endothelial cells with a
combination of TNFα and interferon γ. The down-regulation of CAR
led to reduced transduction efficiency and expression of genes
transferred by Ad. These data suggest that CAR-mediated gene
transfer to ECs using Ads in patients with inflammatory conditions
may exhibit lower efficiency. Ad and AAV transduce vascular
endothelial cells (EC) with low efficiency compared to more
permissive cell types such as heptocytes. This precludes their use for
systemic gene delivery to the vasculature. Dr. Andrew Baker’s group
inserted EC-targeting peptides (identified by phage display) into the
HI loop of Ad serotype 5 fibers containing pre-existing mutations
that block Ad:CAR binding. They also inserted similar peptides into
the capsid of AAV serotype 2. The double mutant Ad and the mutant
AAV-2 both demonstrated markedly increased tropism to EC. These
novel Ad and AAV vectors have important implications for gene
therapy targeted at the vasculature. Finally, Dr. Frank Giordano
explored ways for improving gene transfer to the myocardium using
vectors that are delivered inside the coronary vasculature. He used
peptide display and protein design strategies to develop peptide
sequences that facilitated transendothelial passage of
macromolecules. He infused Ad encoding nuclear-targeted ßgalactosidase into the coronary vasculature of hearts isolated from
CD-1 mice at constant pressure with and without non-covalently
complexed peptide sequences, and then transplanted the hearts into
recipient immuno-deficient SCID mice. Four days later he extracted
the hearts, fixed and stained them with X-gal. Simple non-covalent
pre-complexing with specific peptide sequences increased
intravascular gene delivery to the myocardium by greater than threefold over Ad alone. These experiments represent a novel approach
to in-vivo transduction of the myocardium and other tissues by
intravascular delivery.
The exhibit hall was a popular place for meeting attendees to check out the latest technology
and products.
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MARK
YOUR
ANNUAL MEETING HIGHLIGHTS
C ALENDARS
NOW
FOR
6 th Annual Meeting
June 4-8, 2003
Washington, D.C.
PROGRAM TOPICS WILL FEATURE:
• Concepts in Multi-Gene Therapy
• Gene Therapy in Cooperation with Standard
Medical Practices
• Gene Transfer and Stem Cells
• Vector Targeting and Control of
Transgene Expression
• Right Target, Right Gene,
Right Vector Combinations
• Host Response to Gene Transfer Vehicles
...plus workshops and symposia organized
by the Society’s scientific committees
THE
AMERICAN SOCIETY OF GENE THERAPY
ASGT 5TH ANNUAL MEETING:
PRE CLINICAL
AND CLINICAL
by W. French Anderson, MD
GENE THERAPY
The 2002 ASGT meeting provided an excellent forum for exchanging the
latest information regarding the present and future use of gene therapy
approaches to treat human diseases. Diseases of the Respiratory system
such as asthma are both common and serious in the United States. A
special session describing promising gene therapies for asthma featured
talks by Drs. Joel Kline and Art Krieg. The progress in the development of
gene therapies for respiratory diseases like cystic fibrosis and emphysema
was presented in another special session featuring talks by Drs. Paul
Reynolds and Ray Pickles.
Diseases caused by mutations in single genes have always been considered
prime candidates for gene therapy. As a group, the lysozomal storage
diseases are relatively common and devastating to the families carrying the
mutant genes. In the plenary session, the laboratories of Drs. Mark Haskins
and Kathy Ponder described the treatment and cure of dogs with a severe
inherited lysozomal storage disease by gene therapy. No member of the
audience will forget the video showing the treated dogs running up to and
around their untreated brother. The presentation of these results at the
2002 ASGT Annual Meeting highlighted the outstanding potential and rapid
progress in the development of gene therapy for any number of human
disorders in the near future.
A highlight of the ASGT 2002 meeting was the presentation of several
clinical trials. Most exciting was the ongoing success in treating SCID by
gene therapy. Dr. Claudio Bordignon’s group in Milan reported the
apparent cure of two ADA-deficient SCID patients who, for logistic
reasons, could not be given PEG-ADA. Together with Dr. Don Kohn’s
earlier data, it now appears that some patients with ADA-deficient SCID
can be cured by gene therapy if PEG-ADA is not given. The PEG-ADA
appears to interfere with the positive growth selection of the geneengineered blood cells in the patient.
In addition to Dr. Bordignon’s data, Dr. Fabio Candotti presented the initial
data from the NIH clinical trial where four ADA-deficient SCID patients
thus far have been treated simultaneously with two different retroviral
vectors carrying the ADA gene. At this early stage, it cannot yet be
determined which of the two new vectors is the more successful
construct. Dr. Kohn presented eight year follow-up data on his newborn
umbilical cord stem cell ADA-deficient SCID gene therapy protocol.
Finally, there was follow-up on Dr. Alain Fischer’s successful
treatments of a number of children with γc-deficient SCID.
Dr. Timothy Henry and colleagues presented results from their
clinical trial in which patients with critical limb ischemia appeared to
be significantly helped by the administration of an expression plasmid
containing the human FGF-1 gene. A number of successes in treating
ischemia have been reported in the past by administering the VEGF
gene, but FGF-1 may have certain advantages, such as a reduced
tendency for hemangioma formation.
Dr. Geoff Symonds and colleagues presented their Phase I/II clinical
trial using an anti-tat gene therapy strategy against AIDS. The
approach is to treat the patient’s CD34-positive blood cells ex vivo
with a retroviral vector containing a ribozyme targeting tat. Results
suggested that a Phase II trial would be appropriate.
Hemophilia B is an excellent candidate for gene therapy as the
deficient protein, Factor IX (FIX), is secreted and the levels needed to
correct the disorder are far less than the normal level of Factor IX
meaning that neither high levels of expression nor the introduction of
the FIX gene into a large number of cells should be necessary. The
development of a clinical trial based on the correction of hemophilia
B in both mouse and canine models was discussed by Drs. Katherine
High and Mark Kay. They also reported on the safety and progress of
hemophilia B clinical trials they are conducting.
Two studies about to go into Phase I/II clinical trials were presented.
Dr. Catherine Bollard and colleagues reported successful animal
studies in a gene therapy approach wherein CTL are made TGF- ß
resistant by ex vivo transduction with a retroviral vector carrying a
dominant-negative TGF- ß Type II receptor. Their approach would be
used to treat Hodgkin’s and other TGF- ß secreting cancers.
Finally, Dr. Jedd Wolchok and colleagues presented significant animal
data suggesting that a xenogenic DNA vaccine approach may be
useful in treating malignant melanoma. The canine equivalent of
malignant melanoma was successfully treated with a plasmid carrying
the human tyrosinase gene as a vaccine.
ASGT 5TH ANNUAL MEETING:
GENE THERAPY FOR THE NERVOUS
AND CONNECTIVE TISSUE DISORDERS
by David Bodine, PhD
The Scientific Program of the 2002 ASGT Annual Meeting featured many
novel and exciting pre-clinical models for Gene Therapy. Two workshops
and a scientific symposium were devoted to describing novel methods to
introduce genes into the central nervous system (CNS). The topics
covered included new vectors to improve gene delivery and/or expression
in the CNS by Drs. Jacques Mallet, Thomas Wickham, Pedro Lowenstein,
Nick Mazarakis and David Fink, and methods to improve the distribution
of vectors and proteins in the CNS by Drs. Mark Sands, Beverly Davidson,
William Pardridge, and Krys Bankiewicz. Lectures by Drs. Joe Glorioso,
Xandra Breakefield, Michel Pohl, Michael Chancellor and David Fink
described the use of herpes virus based vectors to introduce genes into
the cells of the peripheral nervous system. Gene transfer is a powerful
tool to mark brain cells for identification after transplantation, and the
potential for therapeutic use of neural stem cells for treatment of neural
disorders such as Parkinson’s disease and Alzheimer’s disease was clear
from the talks presented by Drs. Howard Federoff, Evan Snyder, Bill
Hauswirth and Matthew During.
Musculoskeletal disorders were also recognized as excellent candidates for
gene therapy as well. Drs. Jeff Chamberlain, Louis Kunkel, Thomas Rando
and Kevin Campbell described their pioneering work on gene delivery to
large muscles and their progress towards an eventual gene therapy for
diseases like Muscular Dystrophy. Dr. Paul Robbins chaired a workshop
focused on improving bone and cartilage healing that featured
presentations by Drs. Wim van den berg, Steve Ghivizzani, Dan Gazit and
Axel Baltzer.
5
AMERICAN SOCIETY OF GENE THERAPY
ANNUAL MEETING HIGHLIGHTS
MOLECULAR THERAPY UPDATE
BY INDER VERMA, PHD
EDITOR AND CHIEF
Robert (Rob) Murray Frederickson has been appointed the
Managing Editor of Molecular Therapy. He will take over the
responsibilities previously carried out by Fintan Steele.
Frederickson obtained his PhD in Biochemistry and Cell Biology
in 1994 from McGill University in Montreal, Canada. He then
joined Nature magazine as an assistant biology editor. From 1996
to 1998 Frederickson worked in the laboratory of Stan Fields at
the University of Washington in Seattle as a postdoctoral fellow.
In 1998 he rejoined the Nature group, first as Research Editor for
Nature Biotechnology and then as Senior Editor at Nature Medicine.
In November 2000, he was appointed the Editor and Director of
Content Development of the biotechnology web portal Bio.com,
based in Berkeley, California. He most recently was the Editorial
Manager at LifeSpan Biosciences in Seattle. Frederickson is also a
freelance writer for the Elsevier journal Chemistry and Biology.
He is fluent in English and French and brings substantial
experience in scientific publication. Frederickson will be based at
Elsevier’s offices in San Diego, California. The staff of Elsevier, the
editorial board of Molecular Therapy, and the members of the
American Society of Gene Therapy extend Rob Frederickson a
very warm welcome!
Dr. Eric Lander delivering the Presidential Symposium on “The Human Genome and Beyond”.
ASGT President Malcolm Brenner with the Excellence in Research Award recipients.
From left to right: Eunhee Kim, Anja Ehrhardt, Malcolm Brenner, Celine Bouquet,
David Favre, Kathrin Bernt.
6
WATCH YOUR MAIL FOR
THE ASGT CALL FOR ABSTRACTS
THE ABSTRACT DEADLINE FOR
THE 6TH ANNUAL MEETING
IS FEBRUARY 6, 2003
AMERICAN SOCIETY OF GENE THERAPY
The ASGT Job Bank is now available through the home page of the ASGT
website, and can be accessed by both members and non-members. The Job
Bank includes job postings from both academic institutions and
corporations. If you are interested in placing an ad, the ASGT website also
has a simple and easy online job placement form, featuring reduced rates
for members.
Please visit www.asgt.org to experience this popular feature.
WWW. A S G T. O R G
ASGT OFFICERS AND BOARD OF DIRECTORS
President
Joseph Glorioso, PhD
University of Pittsburgh
School of Medicine
Pittsburgh, PA
President-Elect
Donald Kohn, MD
Childrens Hospital of Los Angeles
Los Angeles, CA
Vice President
Katherine High, MD
Childrens Hospital of Philadelphia
Philadelphia, PA
Secretary
David Bodine, PhD
GMBB/NHGRI
Bethesda, MD
Treasurer
Xandra Breakefield, PhD
Massachusetts General Hospital
Charlestown, MA
JUNE 2002 - JUNE 2003
Board Members
Arthur L. Beaudet, MD
Baylor College of Medicine
Houston, TX
R. Scott McIvor, PhD
University of Minnesota Institute of
Human Genetics
Minneapolis, MN
Barrie Carter, PhD
Targeted Genetics Corp
Seattle, WA
Dusty Miller, PhD
Fred Hutchinson
Cancer Research Center
Seattle, WA
Kenneth Cornetta, MD
Indiana University
Indianapolis, IN
Beverly Davidson, PhD
University of Iowa
Iowa City, IA
Cynthia Dunbar, MD
NHLBI Hematology Branch
Bethesda, MD
Philip D. Noguchi, MD
Food and Drug Administration
Rockville, MD
Jon Wolff, MD
University Of Wisconsin
Madison, WI
Chairman, Advisory Board
Savio L.C. Woo, PhD
Mt Sinai School Of Medicine
New York, NY
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AMERICAN SOCIETY OF GENE THERAPY
ASGT 5TH ANNUAL MEETING SUPPORTERS
SPECIAL THANKS TO THESE ORGANIZATIONS AND COMPANIES WHO HAVE PROVIDED FINANCIAL SUPPORT.
Corporate Symposium
Partial Support of Welcome Reception
Six Excellence in Research Awards
General Meeting Support
General Meeting Support
Twenty Travel Grants
Printing of Abstract Supplement
Abstracts on CD-Rom
Printing of Final Program
Six Travel Grants
Corporate Symposium
Fifteen Travel Grants
General Meeting Support
Excellence in Research Award
Schedule-at-a-Glance
T H A N K
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Y O U