Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy

Silencing of end-joining repair for efficient site-specific gene insertion after TALEN/CRISPR mutagenesis inAedes aegypti

(2015) Sanjay Basu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

(2015) David G. Ousterout et al.   Nature Communications

Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy

(2014) Megan S Keiser et al.   MOLECULAR THERAPY

Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

(2014) Alex Mas Monteys et al.   NUCLEIC ACIDS RESEARCH

AAV9-mediated Expression of a Non-self Protein in Nonhuman Primate Central Nervous System Triggers Widespread Neuroinflammation Driven by Antigen-presenting Cell Transduction

(2013) Lluis Samaranch et al.   MOLECULAR THERAPY

RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

(2013) Megan S. Keiser et al.   NEUROBIOLOGY OF DISEASE

Altered Purkinje cell miRNA expression and SCA1 pathogenesis

(2013) Edgardo Rodriguez-Lebron et al.   NEUROBIOLOGY OF DISEASE

Clinical features, neurogenetics and neuropathology of the polyglutamine spinocerebellar ataxias type 1, 2, 3, 6 and 7

(2013) Udo Rüb et al.   PROGRESS IN NEUROBIOLOGY

Pre-existing Anti–Adeno-Associated Virus Antibodies as a Challenge in AAV Gene Therapy

(2013) Vedell Louis Jeune et al.   Human Gene Therapy Methods

Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

(2012) Richard Grondin et al.   BRAIN

Polyglutamine neurodegeneration: expanded glutamines enhance native functions

(2012) Harry T Orr   CURRENT OPINION IN GENETICS & DEVELOPMENT

Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

(2012) Jie Bu et al.   MOLECULAR THERAPY

Spinocerebellar ataxia type 1 (SCA1): new pathoanatomical and clinico-pathological insights

(2012) U. Rüb et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

(2012) J. Bennett et al.   Science Translational Medicine

In vivo monitoring of recovery from neurodegeneration in conditional transgenic SCA1 mice

(2011) Gülin Öz et al.   EXPERIMENTAL NEUROLOGY

14-3-3 Binding to Ataxin-1(ATXN1) Regulates Its Dephosphorylation at Ser-776 and Transport to the Nucleus

(2011) Shaojuan Lai et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Preclinical Safety of RNAi-Mediated HTT Suppression in the Rhesus Macaque as a Potential Therapy for Huntington's Disease

(2011) Jodi L McBride et al.   MOLECULAR THERAPY

Targeted Genome Editing Across Species Using ZFNs and TALENs

(2011) Andrew J. Wood et al.   SCIENCE

Eight Years of Clinical Improvement in MPTP-Lesioned Primates After Gene Therapy With AAV2-hAADC

(2010) Piotr Hadaczek et al.   MOLECULAR THERAPY

In Vivo RNAi-Mediated α-Synuclein Silencing Induces Nigrostriatal Degeneration

(2010) Oleg S Gorbatyuk et al.   MOLECULAR THERAPY

Pathogenic Mechanisms of a Polyglutamine-mediated Neurodegenerative Disease, Spinocerebellar Ataxia Type 1

(2008) Huda Y. Zoghbi et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Detection of Intact rAAV Particles up to 6 Years After Successful Gene Transfer in the Retina of Dogs and Primates

(2008) Knut Stieger et al.   MOLECULAR THERAPY

Opposing effects of polyglutamine expansion on native protein complexes contribute to SCA1

(2008) Janghoo Lim et al.   NATURE

Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi

(2008) J. L. McBride et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The 14-3-3 proteins: integrators of diverse signaling cues that impact cell fate and cancer development

(2008) Deborah K. Morrison   TRENDS IN CELL BIOLOGY