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.

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 8/07/2021

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 12/01/2013

Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia.

Chen SJ, Sanmiguel J, Lock M, McMenamin D, Draper C, Limberis MP, Kassim SH, Somanathan S, Bell P, Johnston JC, Rader DJ, Wilson JM

Human Gene Therapy. Clinical Development

Publication 12/01/2013

The role of apoptosis in immune hyporesponsiveness following AAV8 liver gene transfer.

Faust SM, Bell P, Zhu Y, Sanmiguel J, Wilson JM

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

Publication 3/01/2012

Long-term restoration of cardiac dystrophin expression in golden retriever muscular dystrophy following rAAV6-mediated exon skipping.

Bish LT, Sleeper MM, Forbes SC, Wang B, Reynolds C, Singletary GE, Trafny D, Morine KJ, Sanmiguel J, Cecchini S, Virag T, Vulin A, Beley C, Bogan J, Wilson JM, Vandenborne K, Kornegay JN, Walter GA, Kotin RM, Garcia L, Sweeney HL

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

Publication 1/01/2010

The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques.

Wang L, Calcedo R, Wang H, Bell P, Grant R, Vandenberghe LH, Sanmiguel J, Morizono H, Batshaw ML, Wilson JM

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