Related references
Note: Only part of the references are listed.Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles
Niclas E. Bengtsson et al.
MOLECULAR THERAPY (2021)
Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
Chady H. Hakim et al.
NATURE COMMUNICATIONS (2021)
Duchenne muscular dystrophy
Dongsheng Duan et al.
NATURE REVIEWS DISEASE PRIMERS (2021)
The promise and challenge of therapeutic genome editing
Jennifer A. Doudna
NATURE (2020)
Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system
Yu Zhang et al.
SCIENCE ADVANCES (2020)
CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors
Dan Wang et al.
CELL (2020)
Correction of muscular dystrophies by CRISPR gene editing
Francesco Chemello et al.
JOURNAL OF CLINICAL INVESTIGATION (2020)
High-Resolution Histological Landscape of AAV DNA Distribution in Cellular Compartments and Tissues following Local and Systemic Injection
Junling Zhao et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors
Andrew V. Anzalone et al.
NATURE BIOTECHNOLOGY (2020)
AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity
Ngoc Tam Tran et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells
Yi-Li Min et al.
SCIENCE ADVANCES (2019)
Questions Answered and Unanswered by the First CRISPR Editing Study in a Canine Model of Duchenne Muscular Dystrophy
Nalinda B. Wasala et al.
HUMAN GENE THERAPY (2019)
Cardiac-Specific Expression of H2-R15 Mini-Dystrophin Normalized All Electrocardiogram Abnormalities and the End-Diastolic Volume in a 23-Month-Old Mouse Model of Duchenne Dilated Cardiomyopathy
Nalinda B. Wasala et al.
HUMAN GENE THERAPY (2018)
AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice
Chady H. Hakim et al.
JCI INSIGHT (2018)
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson et al.
SCIENCE (2016)
In vivo tissue-tropism of adeno-associated viral vectors
Arun Srivastava
CURRENT OPINION IN VIROLOGY (2016)
Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Joe W. McGreevy et al.
DISEASE MODELS & MECHANISMS (2015)
Exclusive skeletal muscle correction does not modulate dystrophic heart disease in the aged mdx model of Duchenne cardiomyopathy
Nalinda B. Wasala et al.
HUMAN MOLECULAR GENETICS (2013)
Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration
B. Bostick et al.
GENE THERAPY (2007)
Non-coding RNAs: lessons from the small nuclear and small nucleolar RNAs
A. Gregory Matera et al.
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2007)
Share Paper
