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Pablo Perez-Pinera et al.
NATURE METHODS (2013)
Genome engineering using the CRISPR-Cas9 system
F. Ann Ran et al.
NATURE PROTOCOLS (2013)
TMC1 and TMC2 Are Components of the Mechanotransduction Channel in Hair Cells of the Mammalian Inner Ear
Bifeng Pan et al.
NEURON (2013)
RNA-Guided Human Genome Engineering via Cas9
Prashant Mali et al.
SCIENCE (2013)
Multiplex Genome Engineering Using CRISPR/Cas Systems
Le Cong et al.
SCIENCE (2013)
Genetics of Hearing and Deafness
Simon Angeli et al.
ANATOMICAL RECORD-ADVANCES IN INTEGRATIVE ANATOMY AND EVOLUTIONARY BIOLOGY (2012)
Evidence-Based Path to Newborn Screening for Duchenne Muscular Dystrophy
Jerry R. Mendell et al.
ANNALS OF NEUROLOGY (2012)
BACE1 Protein Endocytosis and Trafficking Are Differentially Regulated by Ubiquitination at Lysine 501 and the Di-leucine Motif in the Carboxyl Terminus
Eugene L. Kang et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2012)
Dihydrotestosterone-Inducible IL-6 Inhibits Elongation of Human Hair Shafts by Suppressing Matrix Cell Proliferation and Promotes Regression of Hair Follicles in Mice
Mi Hee Kwack et al.
JOURNAL OF INVESTIGATIVE DERMATOLOGY (2012)
Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis
Holly B. Kordasiewicz et al.
NEURON (2012)
Cas9-crRNA ribonucleoprotein complex mediates specific DNA cleavage for adaptive immunity in bacteria
Giedrius Gasiunas et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)
A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity
Martin Jinek et al.
SCIENCE (2012)
Potent and Selective Antisense Oligonucleotides Targeting Single-Nucleotide Polymorphisms in the Huntington Disease Gene/Allele-Specific Silencing of Mutant Huntingtin
Jeffrey B. Carroll et al.
MOLECULAR THERAPY (2011)
A nonsense mutation in Gnat1, encoding the alpha subunit of rod transducin, in spontaneous mouse models of retinal dysfunction
Makoto Miyamoto et al.
EXPERIMENTAL EYE RESEARCH (2010)
Visual Responses in Mice Lacking Critical Components of All Known Retinal Phototransduction Cascades
Annette E. Allen et al.
PLOS ONE (2010)
Incidence and Risk Factors of Arrhythmic Events in Catecholaminergic Polymorphic Ventricular Tachycardia
Meiso Hayashi et al.
CIRCULATION (2009)
Targeted delivery of nanoparticles for the treatment of lung diseases
Shirzad Azarmi et al.
ADVANCED DRUG DELIVERY REVIEWS (2008)
MMEJ repair of double-strand breaks (director's cut): deleted sequences and alternative endings
Mitch McVey et al.
TRENDS IN GENETICS (2008)
CRISPR provides acquired resistance against viruses in prokaryotes
Rodolphe Barrangou et al.
SCIENCE (2007)
Sequence variations in PCSK9, low LDL, and protection against coronary heart disease
JC Cohen et al.
NEW ENGLAND JOURNAL OF MEDICINE (2006)
Full motor recovery despite striatal neuron loss and formation of irreversible amyloid-like inclusions in a conditional mouse model of Huntington's disease
M Díaz-Hernández et al.
JOURNAL OF NEUROSCIENCE (2005)
RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model
SQ Harper et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2005)
Polo-like kinases and oncogenesis
F Eckerdt et al.
ONCOGENE (2005)
Viral and non-viral vectors in gene therapy: Technology development and clinical trials
K Lundstrom et al.
TECHNOLOGY IN CANCER RESEARCH & TREATMENT (2003)
Mutations in PCSK9 cause autosomal dominant hypercholesterolemia
M Abifadel et al.
NATURE GENETICS (2003)
Obesity is associated with macrophage accumulation in adipose tissue
SP Weisberg et al.
JOURNAL OF CLINICAL INVESTIGATION (2003)
Angptl3 regulates lipid metabolism in mice
R Koishi et al.
NATURE GENETICS (2002)
Dominant and recessive deafness caused by mutations of a novel gene, TMC1, required for cochlear hair-cell function
K Kurima et al.
NATURE GENETICS (2002)
Identification of genes that are associated with DNA repeats in prokaryotes
R Jansen et al.
MOLECULAR MICROBIOLOGY (2002)
Medical progress - The hemophilias - From royal genes to gene therapy
PM Mannucci et al.
NEW ENGLAND JOURNAL OF MEDICINE (2001)
Medical progress: Amyotrophic lateral sclerosis.
LP Rowland et al.
NEW ENGLAND JOURNAL OF MEDICINE (2001)
Altered stability of pulmonary surfactant in SP-C-deficient mice
SW Glasser et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2001)
Biological significance of a family of regularly spaced repeats in the genomes of Archaea, bacteria and mitochondria
FJM Mojica et al.
MOLECULAR MICROBIOLOGY (2000)
Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease
A Yamamoto et al.
CELL (2000)