Volume: 6, Issue: 1(2011)
pp. 71-74 DOI: 10.1142/S1568558611000295
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Full Text - Free Access (PDF, 200KB)
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References
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| Title: |
NEW RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV)-BASED THERAPEUTICS REACH PIVOTAL PHASE 2 CLINICAL TRIALS
Invited review article.
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| Author(s): |
CHRISTIAN MUELLER
Department of Pediatrics and Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 016555, USASHALESH KAUSHAL
Department of Pediatrics and Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 016555, USAMARGARET HUMPHRIES
Department of Pediatrics and Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 016555, USATERENCE R. FLOTTE
Corresponding author: University of Massachusetts Medical School, Suite S1-340, 55 Lake Avenue North, Worcester, MA 016555, Tel: (508) 856-2107, Fax: (508) 856-8181.
Department of Pediatrics and Ophthalmology and Gene Therapy Center, University of Massachusetts Medical School, Worcester, MA 016555, USA
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| History: |
Received 3 February 2011
Revised 10 February 2011
Accepted 10 February 2011
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| Abstract: |
This brief review summarizes the design of two phase 2 clinical trials of recombinant adeno-associated virus (rAAV)-based vector that are currently underway. These include a trial of a rAAV2-RPE65 vector for patients with Leber congenital amaurosis and a trial of a rAAV1-alpha-1-antitrypsin(AAT) vector delivered intramuscularly in patients with genetic emphysema due to AAT deficiency. In both cases, the current phase 2 trials were preceded by phase 1 trials indicating a good safety profile and persistent transgene expression. |
| Keywords: |
Gene therapy; adeno-associated virus; clinical trial; emphysema; Leber congenital amaurosis; alpha-1 antitrypsin
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