We invite you to discover Human Gene Therapy Clinical Development, a peer-reviewed journal publishing preclinical research and clinical trial data for gene and cell therapies.

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Publication 10/15/2021

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Hemophilia A.

Greig JA, Smith MK, Nordin JML, Goode T, Chroscinski EA, Buza EL, Schmidt N, Kattenhorn LM, Wadsworth S, Wilson JM

Human Gene Therapy

Publication 8/26/2021

Isolating Natural Adeno-Associated Viruses from Primate Tissues with a High-Fidelity Polymerase.

Wang Q, Nambiar K, Wilson JM

Human Gene Therapy

Publication 8/20/2021

Adeno-Associated Virus Vector-Mediated Expression of Antirespiratory Syncytial Virus Antibody Prevents Infection in Mouse Airways.

Tycko J, Adam VS, Crosariol M, Ohlstein J, Sanmiguel J, Tretiakova AP, Roy S, Worgall S, Wilson JM, Limberis MP

Human Gene Therapy

Publication 8/17/2021

Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model.

Greig JA, Jennis M, Dandekar A, Chorazeczewski JK, Smith MK, Ashley SN, Yan H, Wilson JM

Molecular Genetics And Metabolism

Publication 8/07/2021

An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates.

Zabaleta N, Dai W, Bhatt U, Hérate C, Maisonnasse P, Chichester JA, Sanmiguel J, Estelien R, Michalson KT, Diop C, Maciorowski D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Naninck T, Kahlaoui N, Lemaitre J, Qi W, Hudspeth E, Cucalon A, Dyer CD, Pampena MB, Knox JJ, LaRocque RC, Charles RC, Li D, Kim M, Sheridan A, Storm N, Johnson RI, Feldman J, Hauser BM, Contreras V, Marlin R, Tsong Fang RH, Chapon C, van der Werf S, Zinn E, Ryan A, Kobayashi DT, Chauhan R, McGlynn M, Ryan ET, Schmidt AG, Price B, Honko A, Griffiths A, Yaghmour S, Hodge R, Betts MR, Freeman MW, Wilson JM, Le Grand R, Vandenberghe LH

Cell Host & Microbe

Publication 8/04/2021

Context-Specific Function of the Engineered Peptide Domain of PHP.B.

Martino RA, Fluck EC 3rd, Murphy J, Wang Q, Hoff H, Pumroy RA, Lee CY, Sims JJ, Roy S, Moiseenkova-Bell VY, Wilson JM

Journal Of Virology