University of North Carolina School of Medicine created the
Gene Therapy Center in 1996 with the goal of merging molecular
genetics research with healthcare delivery. The Gene Therapy
Center provides important resources to academic investigators
through two core facilities created to support preclinical
and clinical gene therapy studies. These facilities, the Vector
Core and the Human Applications Laboratories, were created
to ensure that investigators would have promising gene vectors
available in the quality and quantities needed for preclinical
or clinical studies. Research in the laboratory has centered
on the molecular biology of adeno-associated virus (AAV) in
order to exploit the unique features of this virus to develop
an efficient viral vector system for use in human gene therapy.
Continued efforts in understanding the mechanism of viral
replication and integration for both wild-type and recombinant
AAV are being pursued in order to create more efficient gene
transfer vectors. The ultimate goal of the Gene Therapy Center
is to facilitate the progression and translation of gene therapy
research from the laboratory bench into Phase I clinical trials
for the treatment of human disease.
Click here to learn more about our
gene therapy research.
Symposium: The Nexus of Gene Therapy & Regenerative Medicine
Location: The Old Salem Visitor Center, Winston-Salem, NC
Date: Thursday, February 7, 2013 9AM - 6PM
Gene Therapists Celebrate a Decade of Progress
FDA Supports Rare Diseases Day
Duchenne muscular dystrophy: Charlie's Story
Steven Gray and his wife Christy attended the Hope and Love Ball in
Albany, NY on February 12. This was a fundraiser for Hannah’s Hope
Fund, an organization
supporting Dr. Gray’s research at the UNC Gene Therapy Center to
develop a treatment for Giant Axonal Neuropathy (GAN). Among the 350
attendees was Doris Buffet, sister of billionaire Warren Buffet and
founder of the Sunshine Lady Foundation, who had previously
donated $500,000 to Hannah’s Hope Fund. Also in attendance was Hannah,
the 6-year-old namesake for Hannah’s Hope. Drs. Steven Gray and Maria
Escolar (UNC Gene Therapy Center) are spearheading an international
effort to study and treat GAN. GAN is an orphan
disease caused by a loss of the gigaxonin gene, and it is characterized
by motor and sensory system degeneration that is terminal by the 3rd decade of life.
Christy Gray, Hannah and Steve Gray Steve Gray and Doris Buffet
October 2010 - Dr. Maria Escolar continues her outstanding research. Click here to learn more.
October 2010 - UNC researcher making great strides in
Duchenne muscular dystrophy (DMD). UNC researchers get glimpse into muscle disorder :: WRAL.com
October 2010 - Richard J. Samulski and the Gene Therapy Center are making exciting findings on
Duchenne muscular dystrophy (DMD). Click here to learn more.
October 2010 - Maria Escolar and the Gene Therapy Center are focusing on treatments for Krabbe Disease. Please click here for Emma Daniels inspirational story!
Gene Therapy PI You Tube!
Joe Kornegay was interviewed at the American College of Veterinary
Internal Medicine (ACVIM) in Seattle last June. The context
within ACVIM is that their work is an example of "One Medicine"
or "Comparative Medicine" intended to advance the understanding
of diseases in both humans and animals.
for Muscular Dystrophy!
join UNC's Air Force ROTC on Thursday, October 15
for a Push-Up-A-Thon to help raise money for MD research.
The event will be held at 4 pm in front of The Center for
Dramatic Art. All proceeds will go to the Senator Paul
Wellstone Muscular Dystrophy Research Center to support MD
research. If you'd like to help sponsor this event, please
send an email to firstname.lastname@example.org.
2009 - The UNC Gene Therapy
Center was featured in the August
30 edition of Parade magazine. Parade published
the article in anticipation of the annual Jerry Lewis Muscular
Dystrophy Association Telethon, which commences Sunday, September
Samulski was elected vice president of the American Society
of Gene and Cell Therapy. This is a three year cycle - one
year as Vice President (2009-2010), then one year as President-Elect
(2010-2011), and a third year as President (2011-2012). He
will be President during the 15th Annual Meeting of ASGCT
taking place in Philadelphia, PA May 16 - 20, 2012.
UNC Senator Paul D. Wellstone
Center of Excellence for Muscular Dystrophy Research held
its inauguration event on March 2, 2009. Distinguished
guests included Rep. David Price and Chancellor Holden Thorpe.
Click here for the UNC press release.
2008 - UNC Gene Therapy Center has
been awarded a five-year, $7 million grant to establish a
muscular dystrophy research center named in honor of the late
Sen. Paul D. Wellstone of Minnesota, who earned his undergraduate
and doctoral degrees at UNC. Click
here for the official press release
2008 - Jude Samulski, Director, has
received the inaugural Oustanding Achievement Award given
by the American Society of Gene Therapy. The award recognizes
an active member of the society who has achieved a pioneering
research success, a specific high-impact accomplishment or
a lifetime of significant contributions to the field of gene
therapy. Click here for the official press release.
- The first clinical
trial for Duchenne Muscular Dystrophy begins, utilizing AAV
vectors developed and produced in Dr. Samulski's laboratory.
Click here for more information.
- The Gene Therapy Center would thank Dr. Mark
Atkinson for speaking at the First Annual Diabetes Research
Seminar to Memorialize Jerri Coleman.
- In memory of Jerri Coleman, the Gene Therapy
Center has established a memorial fund for diabetes
research in her name. Click here
for more information.
January 2005 - Dr. Samulski is interviewed for the MDA's Quest Magazine - "Bridge Over Troubled Waters: Crossing to Safety and Efficacy in Gene Therapy is Current Challenge"
2004 - Drs. Stilwell and Samulski
published a breakthrough study on the safety of AAV virion
shells. They found that viral capsids may influence gene expression
independent from their packaged DNA. AAV and adenovirus were
compared using DNA microarrays for their effects on cellular
gene expression. AAV was found to have minimal impact on gene
expression in the cell, while adenovirus activated the expression
of genes associated with toxicity and the immune response.
These findings indicate that AAV may be a safer and more effective
form of transduction.
Click here for more information about this study.