Sage Journals: Discover world-class research

Retricted access

Research article

First published 2022-5

Allele-specific silencing by RNAi of R92Q and R173W mutations in cardiac troponin T

Loredana Migliore, Federico Galvagni, Enrico Pierantozzi [...]

Experimental Biology and Medicine

Retricted access

Research article

First published 2008-07

Identification and Allele-Specific Silencing of the Mutant Huntingtin Allele in Huntington's Disease Patient-Derived Fibroblasts

P.H.J. van Bilsen, L. Jaspers, M.S. Lombardi [...]

Human Gene Therapy

Free access

Research article

First published 2016-4

Allele-Specific Reduction of the Mutant Huntingtin Allele Using Transcription Activator-Like Effectors in Human Huntington's Disease Fibroblasts

Kyle D. Fink, Peter Deng, Josh Gutierrez [...]

Cell Transplantation

Retricted access

Research article

First published 2018-02

Allele-Specific CRISPR-Cas9 Genome Editing of the Single-Base P23H Mutation for Rhodopsin-Associated Dominant Retinitis Pigmentosa

Pingjuan Li, Benjamin P. Kleinstiver, Mihoko Y. Leon [...]

The CRISPR Journal

Retricted access

Research article

First published 2013-11

Increased Steady-State Mutant Huntingtin mRNA in Huntington's Disease Brain

Wanzhao Liu, Joanna Chaurette, Edith L. Pfister [...]

Journal of Huntington's Disease

Retricted access

Research article

First published 2024-04

RNA Interference Effectors Selectively Silence the Pathogenic Variant GNAO1 c.607 G > A In Vitro

Natalia V. Klementieva, Evgenii A. Lunev, Anna A. Shmidt [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 1999-12

A Mutation-Independent Therapeutic Strategem for Osteogenesis Imperfecta

Sophia Millington-Ward, Brian O'neill, Anna-Sophia Kiang [...]

Nucleic Acid Therapeutics

Retricted access

Research article

First published 2007-10

Rapid and Stable Knockdown of an Endogenous Gene in Retinal Pigment Epithelium

Daniel M. Paskowitz, Kenneth P. Greenberg, Douglas Yasumura [...]

Human Gene Therapy

Free access

Research article

First published 2023-05-02

Facioscapulohumeral Disease as a myodevelopmental disease: Applying Ockham’s razor to its various features

G.W. Padberg, B.G.M. van Engelen, N.C. Voermans

Journal of Neuromuscular Diseases

Retricted access

Research article

First published 2012-9

Rationale for the Development of 2-Aminobenzamide Histone Deacetylase Inhibitors as Therapeutics for Friedreich Ataxia

Elisabetta Soragni, Chunping Xu, Heather L. Plasterer [...]

Journal of Child Neurology

Search results