Molecular Therapy
Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy.
Duan H, Zhang C, Zhang Z, Wang X, Zhang J, Yang L, He F, Mao L, Yang L, Pan Z, Yin F, Han R, Wang W, Pan D, Xiao W, Peng J.
2025
The research in the Han Lab is directed to understand the molecular mechanisms of genetic diseases affecting the neuromuscular system, and to develop novel therapeutic strategies to combat these devastating diseases.
Gene therapy with covalently closed-end AAV vector for spinal muscular atrophy.
Duan H, Zhang C, Zhang Z, Wang X, Zhang J, Yang L, He F, Mao L, Yang L, Pan Z, Yin F, Han R, Wang W, Pan D, Xiao W, Peng J.
2025
Recent advances in therapeutic gene editing technologies.
Liu D, Cao D, Han R.
2025
Promotion or inhibition? This is a question in gene editing
Tsai L-K, Han R, Yang D, Chen YE, Zhang J, Xu J
2025
Rapidly Evolving Genome and Epigenome Editing Technologies
Tran NT, Han R.
2024
Systemic Delivery of Full-Length Dystrophin in Duchenne Muscular Dystrophy Mice.
Zhou R, Zhang C, Xiao W, Herzog RW, Han R.
2024
Hit-and-run epigenome editing durably lowers cholesterol in mice.
Han R.
2024
Defective BVES-mediated feedback control of cAMP in muscular dystrophy.
Li H, Wang P, Zhang C, Zuo Y, Zhou Y, Han R.
2023
Systemic AAV9.BVES Delivery Ameliorates Muscular Dystrophy in a Mouse Model of LGMDR25.
Li H, Wang P, Hsu E, Pinckard KM, Stanford KI and Han R.
2022
Efficient precise in vivo base editing in adult dystrophic mice.
Xu L, Zhang C, Li H, Wang P, Gao Y, Mokadam NA, Ma J, Arnold WD, Han R.
2021
Genome-wide CRISPR screen identifies LGALS2 as an oxidative stress-responsive gene with an inhibitory function on colon tumor growth.
Li H, Zhao L, Lau YS, Zhang C, Han R.
2020
BEON: A functional fluorescence reporter for quantification and enrichment of adenine base editing activity.
Wang P, Xu L, Gao Y and Han R.
2020
Life-long AAV-mediated CRISPR genome editing in dystrophic heart improves cardiomyopathy without causing serious lesions in mdx mice.
Xu L#, Lau YS#, Gao Y#, Li H, Han R.
2019
In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
El Refaey M, Xu L, Gao Y, Canan BD, Adesanya TMA, Warner SC, Akagi K, Symer DE, Mohler PJ, Ma J, Janssen PML, Han R.
2017
CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice.
Xu L, Park KH, Zhao L, Xu J, El Refaey M, Gao Y, Zhu H, Ma J, Han R.
2015
The intracellular Ca2+ channel MCOLN1 is required for sarcolemma repair to prevent muscular dystrophy.
Cheng X, Zhang X, Gao Q, Ali Samie M, Azar M, Tsang WL, Dong L, Sahoo N, Li X, Zhuo Y, Garrity AG, Wang X, Ferrer M, Dowling J, Xu L, Han R, Xu H.
2014
Mitochondrial cardiolipin is required for Nlrp3 inflammasome activation.
Iyer SS, He Q, Janczy JR, Elliott EI, Zhong Z, Olivier AK, Sadler JJ, Knepper-Adrian V, Han R, Qiao L, Eisenbarth SC, Nauseef WM, Cassel SL, Sutterwala FS.
2013
TRPM2 links oxidative stress to NLRP3 inflammasome activation.
Zhong Z, Zhai Y, Liang S, Mori Y, Han R, Sutterwala FS, Qiao L.
2013
Genetic ablation of complement C3 attenuates muscle pathology in dysferlin-deficient mice.
Han R, Frett EM, Levy JR, Rader EP, Lueck JD, Bansal D, Moore SA, Ng R, Beltran-Valero de Bernabe D, Faulkner JA, Campbell KP.
2010
Basal lamina strengthens cell membrane integrity via the laminin G domain-binding motif of alpha-dystroglycan.
Han R, Kanagawa M, Yoshida-Moriguchi T, Rader EP, Ng RA, Michele DE, Muirhead DE, Kunz S, Moore SA, Iannaccone ST, Miyake K, McNeil PL, Mayer U, Oldstone MB, Faulkner JA, Campbell KP.
2009
Dysferlin-mediated membrane repair protects the heart from stress-induced left ventricular injury.
Han R, Bansal D, Miyake K, Muniz VP, Weiss RM, McNeil PL, Campbell KP.
2007