Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results. - Wikidata - wikimedia - TriplyDB

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.

http://www.wikidata.org/entity/Q35496285

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Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B Manufacturing of recombinant adeno-associated viral vectors for clinical trials Adeno-associated virus-mediated cancer gene therapy: current status Challenges and Prospects for Alpha-1 Antitrypsin Deficiency Gene Therapy Gene therapy for hemophilia Downstream processing and chromatography based analytical methods for production of vaccines, gene therapy vectors, and bacteriophages Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection Barriers to inhaled gene therapy of obstructive lung diseases: A review Passive immunization against HIV/AIDS by antibody gene transfer.Intranasal antibody gene transfer in mice and ferrets elicits broad protection against pandemic influenza.Sustained normalization of neurological disease after intracranial gene therapy in a feline model.Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.Attenuation of monocrotaline-induced pulmonary hypertension by luminal adeno-associated virus serotype 9 gene transfer of prostacyclin synthase Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Gene Therapy 2017: Progress and Future Directions.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial.Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold?Alpha-1 proteinase inhibitors for the treatment of alpha-1 antitrypsin deficiency: safety, tolerability, and patient outcomes.Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression.Intravenous AAV8 Encoding Urocortin-2 Increases Function of the Failing Heart in Mice.The AAV vector toolkit: poised at the clinical crossroads Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.Reprogramming Immune Response With Capsid-Optimized AAV6 Vectors for Immunotherapy of Cancer Robust Lentiviral Gene Delivery But Limited Transduction Capacity of Commonly Used Adeno-Associated Viral Serotypes in Xenotransplanted Human Skin.Recombinant AAV Vectors for Enhanced Expression of Authentic IgG.Cell and gene therapy for genetic diseases: inherited disorders affecting the lung and those mimicking sudden infant death syndrome AAV-based RNAi silencing of NADPH oxidase gp91(phox) attenuates cold-induced cardiovascular dysfunction Strategies to circumvent humoral immunity to adeno-associated viral vectors.Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level.Alleviation of lung inflammatory responses by adeno-associated virus 2/9 vector carrying CC10 in OVA-sensitized mice Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia Transgenic models of Alzheimer's disease: better utilization of existing models through viral transgenesis.CpG-depleted adeno-associated virus vectors evade immune detection.Immune responses to AAV vectors: overcoming barriers to successful gene therapy

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P2860

Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results.

http://www.wikidata.org/entity/Q35496285

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2011 nî lūn-bûn

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2011 թուականի Օգոստոսին հրատարակուած գիտական յօդուած

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2011 թվականի օգոստոսին հրատարակված գիտական հոդված

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2011年の論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年論文

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2011年论文

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Phase 2 clinical trial of a re ...... -antitrypsin: interim results.

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Human Gene Therapy

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Phase 2 clinical trial of a re ...... -antitrypsin: interim results.

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Anthony T Yachnis

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Brenna Carey

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Bruce C Trapnell

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Christian Mueller

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David R Knop

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Farshid Rouhani

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Guo-jie Ye

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Jeffrey D Chulay

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Louis M Messina

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Margaret Humphries

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P2860

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer.

Expression of human alpha1-antitrypsin in mice and dogs following AAV6 vector-mediated gene transfer to the lungs.

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

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle.

Preclinical evaluation of a recombinant adeno-associated virus vector expressing human alpha-1 antitrypsin made using a recombinant herpes simplex virus production method

Clinical practice. Alpha1-antitrypsin deficiency.

A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

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1239-1247

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scholarly article

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10.1089/HUM.2011.053

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10

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22

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Martha Campbell-Thompson

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2011-08-24T00:00:00Z

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51161968

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21609134

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phase II clinical trial

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3205788

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