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Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

UNC Libraries(2020)10.17615/fzyc-s786Source: DataRank Database

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial is a research paper published in UNC Libraries (2020). On theSindex it has a DataRank of 0.

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Enrichment:Funding (4 grants)OA: hybridTopics (24)IDs (PubMed)SDGs (1)

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Authors (16)

Tong Gui,Scott McPhee,Carmen J. Booth ORCID,Junjiang Sun ORCID,Tal Kafri ORCID

Live enrichment

PubMed:19910364

National Institutes of Health

Grant: 5R01DK058702-12

Nonintegrating Lentiviral Vectors Towards Clinical Trials

National Institutes of Health

Grant: 5P01HL112761-03

ANIMAL MODELS CORE

National Institutes of Health

Grant: 1RC3HL103396-01

Self-Complementary AAV2 Factor IX for Hemophilia B

National Institutes of Health

Grant: 5P01HL066973-05

Gene Therapy Pulmonary &Hematologic Disorders

hybridLicense: CC BY

https://doi.org/10.1089/hum.2014.106

https://www.liebertpub.com/doi/pdf/10.1089/hum.2014.106

https://dx.doi.org/10.17615/fzyc-s786

https://pubmed.ncbi.nlm.nih.gov/25419787

http://dx.doi.org/10.1089/hum.2014.106

Fields of Study

0301 basic medicine0303 health sciences03 medical and health sciences

Keywords

MaleMice, KnockoutClinical Trials as TopicGenetic VectorsDrug Evaluation, PreclinicalGene ExpressionHemorrhageGenetic TherapyDependovirusAntibodies, NeutralizingHemophilia BFactor IXMice, Inbred C57BLDisease Models, AnimalMiceCapsidLiverAnimalsHumansGenetic EngineeringResearch Articles

Sustainable Development Goals

SDG 3: 3. Good health