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 10/04/2022

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors.

Horiuchi M, Hinderer CJ, Greig JA, Dyer C, Buza EL, Bell P, Chichester JA, Hayashi PM, Yan H, Goode T, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 7/29/2022

Characterizing Complex Populations of Endogenous Adeno-associated Viruses by Single-Genome Amplification.

Nambiar K, Wang Q, Yan H, Wilson JM

Human Gene Therapy

Publication 5/10/2022

Durable immunogenicity, adaptation to emerging variants and low dose efficacy of AAV-based COVID19 platform in macaques.

Zabaleta N, Bhatt U, Hérate C, Maisonnasse P, Sanmiguel J, Diop C, Castore S, Estelien R, Li D, Dereuddre-Bosquet N, Cavarelli M, Gallouët AS, Pascal Q, Naninck T, Kahlaoui N, Lemaitre J, Relouzat F, Ronzitti G, Thibaut HJ, Montomoli E, Wilson JM, Le Grand R, Vandenberghe LH

Molecular Therapy : The Journal Of The American Society Of Gene Therapy

Publication 3/22/2022

Efficacy and Safety of a Krabbe Disease Gene Therapy.

Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM

Human Gene Therapy

Publication 1/19/2022

Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression.

Wang L, Warzecha CC, Kistner A, Chichester JA, Bell P, Buza EL, He Z, Pampena MB, Couthouis J, Sethi S, McKeever K, Betts MR, Kakkis E, Wilson JM, Wadsworth S, Sullivan BA

Molecular Therapy. Methods & Clinical Development

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