Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results-Reference-Cited by-同舟云学术

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Phase 2 Clinical Trial of a Recombinant Adeno-Associated Viral Vector Expressing α1-Antitrypsin: Interim Results

Published:2011-10 Issue:10 Volume:22 Page:1239-1247
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Flotte Terence R.¹,Trapnell Bruce C.²,Humphries Margaret¹,Carey Brenna²,Calcedo Roberto³,Rouhani Farshid⁴,Campbell-Thompson Martha⁴,Yachnis Anthony T.⁴,Sandhaus Robert A.⁵,McElvaney Noel G.⁶,Mueller Christian¹,Messina Louis M.¹,Wilson James M.³,Brantly Mark⁴,Knop David R.⁷,Ye Guo-jie⁷,Chulay Jeffrey D.⁷

Affiliation:

1. University of Massachusetts Medical School, Worcester, MA 01655.

2. Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229.

3. University of Pennsylvania, Philadelphia, PA 19104.

4. University of Florida School of Medicine, Gainesville, FL 32610.

5. National Jewish Health, Denver, CO 80206.

6. Beaumont Hospital, Dublin, 9 Ireland.

7. Applied Genetic Technologies Corporation, Alachua, FL 32615.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2011.053

Reference28 articles.

1. Reengineering a receptor footprint of adeno-associated virus enables selective and systemic gene transfer to muscle

2. Use of a Highly Purified α1-Antitrypsin Standard to Establish Ranges for the Common Normal and Deficient α1-Antitrypsin Phenotypes

3. Phase I Trial of Intramuscular Injection of a Recombinant Adeno-Associated Virus Serotype 2α1-Antitrypsin (AAT) Vector in AAT-Deficient Adults

4. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

5. Several Log Increase in Therapeutic Transgene Delivery by Distinct Adeno-Associated Viral Serotype Vectors

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1. Limb Perfusion Delivery of a rAAV1 Alpha-1 Antitrypsin Vector in Non-Human Primates Is Safe but Insufficient for Therapy;Genes;2024-09-10

2. Drug‐induced liver injury related to gene therapy: A new challenge to be managed;Liver International;2024-09-09

3. Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies;Molecular Therapy - Methods & Clinical Development;2024-09

4. Role of FoxP3⁺ Regulatory T Cells in Modulating Immune Responses to Adeno-Associated Virus Gene Therapy;Human Gene Therapy;2024-07-01

5. Miniaturization of hiPSC-derived 3D neural cultures in stirred-tank bioreactors for parallelized preclinical assessment of rAAV;Frontiers in Bioengineering and Biotechnology;2024-04-26

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