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 7/15/2021

Intranasal gene therapy to prevent infection by SARS-CoV-2 variants.

Sims JJ, Greig JA, Michalson KT, Lian S, Martino RA, Meggersee R, Turner KB, Nambiar K, Dyer C, Hinderer C, Horiuchi M, Yan H, Huang X, Chen SJ, Wilson JM

Plos Pathogens

Publication 6/30/2021

Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device.

Shepherd SJ, Warzecha CC, Yadavali S, El-Mayta R, Alameh MG, Wang L, Weissman D, Wilson JM, Issadore D, Mitchell MJ

Nano Letters

Publication 5/05/2021

Developing a second-generation clinical candidate AAV vector for gene therapy of familial hypercholesterolemia.

Wang L, Muthuramu I, Somanathan S, Zhang H, Bell P, He Z, Yu H, Zhu Y, Tretiakova AP, Wilson JM

Molecular Therapy. Methods & Clinical Development

Publication 2/21/2021

Helper lipid structure influences protein adsorption and delivery of lipid nanoparticles to spleen and liver.

Zhang R, El-Mayta R, Murdoch TJ, Warzecha CC, Billingsley MM, Shepherd SJ, Gong N, Wang L, Wilson JM, Lee D, Mitchell MJ

Biomaterials Science

Publication 2/18/2021

Long-term stable reduction of low-density lipoprotein in nonhuman primates following in vivo genome editing of PCSK9.

Wang L, Breton C, Warzecha CC, Bell P, Yan H, He Z, White J, Zhu Y, Li M, Buza EL, Jantz D, Wilson JM

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

Publication 1/07/2021

CRISPR/Cas9 directed to the Ube3a antisense transcript improves Angelman syndrome phenotype in mice.

Schmid RS, Deng X, Panikker P, Msackyi M, Breton C, Wilson JM

The Journal Of Clinical Investigation