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

Publication 12/25/2020

Increasing the Specificity of AAV-Based Gene Editing through Self-Targeting and Short-Promoter Strategies.

Breton C, Furmanak T, Avitto AN, Smith MK, Latshaw C, Yan H, Greig JA, Wilson JM

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

Publication 11/11/2020

MicroRNA-mediated inhibition of transgene expression reduces dorsal root ganglion toxicity by AAV vectors in primates.

Hordeaux J, Buza EL, Jeffrey B, Song C, Jahan T, Yuan Y, Zhu Y, Bell P, Li M, Chichester JA, Calcedo R, Wilson JM

Science Translational Medicine

Publication 10/12/2020

A single injection of an optimized AAV vector into cerebrospinal fluid corrects neurological disease in a murine model of GM1 gangliosidosis.

Hinderer C, Nosratbakhsh B, Katz N, Wilson JM

Human Gene Therapy