[Bionews] Human Gene Therapy

Masi, Paula via bionews%40net.bio.net (by PMasi At liebertpub.com)
Thu Dec 7 11:56:31 EST 2006

For immediate release


Contact: Vicki Cohn, Mary Ann Liebert, Inc, (914) 740-2100, ext. 2156,
vcohn At liebertpub.com 

Safety and Potential Efficacy of Gene Therapy to Treat Genetic Disorder
in Humans Is Described in Human Gene Therapy
New Rochelle, NY, December 7, 2006-Promising early-stage results of a
gene therapy trial to replace Alpha-1 Antitrypsin (AAT) offer hope for
the estimated 100,000 Americans with this life-threatening genetic form
of emphysema, according to a paper published online ahead of print in
the December 2006 issue (Volume 17, Number 12) of Human Gene Therapy, a
peer-reviewed journal published by Mary Ann Liebert, Inc. The paper is
available free online at www.liebertpub.com/hum
            AAT deficiency is one of the three most prevalent fatal
genetic disorders. The other two are cystic fibrosis and Down's
syndrome. The goal of gene therapy, or gene replacement, is to enable
affected individuals to produce the alpha1 antitrypsin protein,
eliminating the need for costly and difficult protein replacement
therapy that requires weekly intravenous injections. 
            Studies in animals have suggested that periodic
intramuscular injections of the AAT gene packaged in a viral delivery
vector could result in safe, long-lasting production of recombinant AAT
protein at therapeutic levels. 
            In this Phase I trial of AAT gene therapy, Brantly, Flotte
and colleagues at the University of Florida, Gainesville, injected
increasing doses of recombinant adeno-associated virus serotype 2
(rAAV2)-AAT vector into 12 AAT-deficient adults. All subjects
discontinued AAT protein replacement therapy 28 days before receiving
gene therapy. 
            The authors reported no serious adverse effects related to
the gene therapy vector, supporting its safety. They were able to detect
viral DNA sequences in the patients' blood, as well as antibodies to the
viral vector. No other immune response to the recombinant vector was
            In the paper entitled, "Phase I Trial of Intramuscular
Injection of a Recombinant Adeno-associated Virus Serotype 2 (rAAV2)
Alpha-1 Antitrypsin (AAT) Vector in AAT-deficient Adults," Brantly et al
report detecting low levels of AAT protein in the blood of one patient
who had not been on AAT replacement therapy before the trial. Residual
levels of previously injected AAT would likely have masked the presence
of gene therapy-generated protein in other study subjects.
            "This study represents an important first step in
establishing the safety of gene replacement therapy for an important
genetic disease. Expression of recombinant protein was low although
there are a number of strategies that can be pursued to improve upon it
in subsequent studies," says James M. Wilson, M.D., Ph.D.,
Editor-in-Chief, and Head of the Gene Therapy Program, Department of
Pathology and Laboratory Medicine, University of Pennsylvania School of
Medicine, in Philadelphia.
Human Gene Therapy is an authoritative peer-reviewed journal published
monthly in print and online that presents reports on the transfer and
expression of genes in mammals, including humans. Related topics include
improvements in vector development, delivery systems, and animal models,
particularly in the areas of cancer, heart disease, viral disease,
genetic disease, and neurological disease, as well as ethical, legal,
and regulatory issues related to the gene transfer in humans. Tables of
contents and a free sample issue may be viewed online at
www.liebertpub.com <http://www.liebertpub.com/> 
            Mary Ann Liebert, Inc., is a privately held, fully
integrated media company known for establishing authoritative
peer-reviewed journals in many promising areas of science and biomedical
research, including Tissue Engineering, Stem Cells and Development, and
Cloning and Stem Cells. Its biotechnology trade magazine, Genetic
Engineering News (GEN), was the first in its field and is today the
industry's most widely read publication worldwide. A complete list of
the firm's 60 journals, books, and newsmagazines is available at
www.liebertpub.com <http://www.liebertpub.com/> .
Mary Ann Liebert, Inc.    140 Huguenot St., New Rochelle, NY 10801-5215
www.liebertpub.com <http://www.liebertpub.com/> 
                                  Phone: (914) 740-2100    (800)
M-LIEBERT    Fax:  (914) 740-2101

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