Allele-Specific Silencing of Mutant Myh6 Transcripts in Mice Suppresses Hypertrophic Cardiomyopathy

A systematic review and meta-analysis of genotype–phenotype associations in patients with hypertrophic cardiomyopathy caused by sarcomeric protein mutations

(2013) Luís R Lopes et al.   HEART

Single-Stranded RNAs Use RNAi to Potently and Allele-Selectively Inhibit Mutant Huntingtin Expression

(2012) Dongbo Yu et al.   CELL

Hypertrophic Cardiomyopathy in 2012

(2012) Carolyn Y. Ho   CIRCULATION

Hypertrophic cardiomyopathy: Translating cellular cross talk into therapeutics

(2012) Polakit Teekakirikul et al.   JOURNAL OF CELL BIOLOGY

Hypertrophic cardiomyopathy

(2012) Barry J Maron et al.   LANCET

Unequal allelic expression of wild-type and mutated β-myosin in familial hypertrophic cardiomyopathy

(2011) Snigdha Tripathi et al.   BASIC RESEARCH IN CARDIOLOGY

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution

(2010) K-M R Prasad et al.   GENE THERAPY

Cardiac fibrosis in mice with hypertrophic cardiomyopathy is mediated by non-myocyte proliferation and requires Tgf-β

(2010) Polakit Teekakirikul et al.   JOURNAL OF CLINICAL INVESTIGATION

Sudden Deaths in Young Competitive Athletes

(2009) Barry J. Maron et al.   CIRCULATION

Adeno-Associated Virus (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat

(2008) Lawrence T. Bish et al.   HUMAN GENE THERAPY