4.6 Article

A Selective Alkylating Agent for CTG Repeats in Myotonic DystrophyType 1

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Article Medicine, Research & Experimental

Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

Beatrice Cardinali et al.

Summary: CRISPR/Cas9-mediated therapeutic gene editing shows promise in treating monogenic diseases like myotonic dystrophies. Recent studies have successfully applied this technology to delete the pathogenic CTG-repeat expansion in the DMPK gene, leading to beneficial effects on disease hallmarks. An approach using specific proguide RNAs and a dual-vector system has enabled efficient and inducible gene editing in cells and tissues relevant to myotonic dystrophy.

MOLECULAR THERAPY-NUCLEIC ACIDS (2022)

Article Genetics & Heredity

A slipped-CAG DNA-binding small molecule induces trinucleotide-repeat contractions in vivo

Masayuki Nakamori et al.

NATURE GENETICS (2020)

Article Medicine, Research & Experimental

Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach

Miki Ikeda et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)

Review Biotechnology & Applied Microbiology

Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors

Andrew V. Anzalone et al.

NATURE BIOTECHNOLOGY (2020)

Article Multidisciplinary Sciences

Precise small-molecule cleavage of an r(CUG) repeat expansion in a myotonic dystrophy mouse model

Alicia J. Angelbello et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Multidisciplinary Sciences

Intrinsically cell-penetrating multivalent and multitargeting ligands for myotonic dystrophy type 1

JuYeon Lee et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Biochemistry & Molecular Biology

Precise small-molecule recognition of a toxic CUG RNA repeat expansion

Suzanne G. Rzuczek et al.

NATURE CHEMICAL BIOLOGY (2017)

Article Biotechnology & Applied Microbiology

Non-coding RNAs as drug targets

Masayuki Matsui et al.

NATURE REVIEWS DRUG DISCOVERY (2017)

Review Clinical Neurology

Genome engineering: a new approach to gene therapy for neuromuscular disorders

Christopher E. Nelson et al.

NATURE REVIEWS NEUROLOGY (2017)

Article Medicine, Research & Experimental

CRISPR/Cas9-Mediated Deletion of CTG Expansions Recovers Normal Phenotype in Myogenic Cells Derived from Myotonic Dystrophy 1 Patients

Claudia Provenzano et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2017)

Review Clinical Neurology

The prospects of CRISPR-based genome engineering in the treatment of neurodegenerative disorders

Jun Wan Shin et al.

THERAPEUTIC ADVANCES IN NEUROLOGICAL DISORDERS (2017)

Article Genetics & Heredity

Disease-associated repeat instability and mismatch repair

Monika H. M. Schmidt et al.

DNA REPAIR (2016)

Review Biochemistry & Molecular Biology

The Emerging Role of RNA as a Therapeutic Target for Small Molecules

Colleen M. Connelly et al.

CELL CHEMICAL BIOLOGY (2016)

Review Pharmacology & Pharmacy

X-ray crystallography over the past decade for novel drug discovery - where are we heading next?

Heping Zheng et al.

EXPERT OPINION ON DRUG DISCOVERY (2015)

Article Chemistry, Multidisciplinary

Rationally Designed Small Molecules That Target Both the DNA and RNA Causing Myotonic Dystrophy Type 1

Lien Nguyen et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2015)

Article Biochemistry & Molecular Biology

Molecular mechanisms in DM1-a focus on foci

Olof Joakim Pettersson et al.

NUCLEIC ACIDS RESEARCH (2015)

Article Biochemistry & Molecular Biology

Reducing Levels of Toxic RNA with Small Molecules

Leslie A. Coonrod et al.

ACS CHEMICAL BIOLOGY (2013)

Article Biochemistry & Molecular Biology

Length-dependent CTG.CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells

Jintang Du et al.

HUMAN MOLECULAR GENETICS (2013)

Review Biochemistry & Molecular Biology

Repeat-associated non-ATG (RAN) translation in neurological disease

John D. Cleary et al.

HUMAN MOLECULAR GENETICS (2013)

Review Clinical Neurology

Myotonic dystrophy: is a narrow focus obscuring the rest of the field?

Mani S. Mahadevan

CURRENT OPINION IN NEUROLOGY (2012)

Article Biochemistry & Molecular Biology

Best practice guidelines and recommendations on the molecular diagnosis of myotonic dystrophy types 1 and 2

Erik-Jan Kamsteeg et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2012)

Article Multidisciplinary Sciences

Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Thurman M. Wheeler et al.

NATURE (2012)

Article Multidisciplinary Sciences

RNase H-mediated degradation of toxic RNA in myotonic dystrophy type 1

Johanna E. Lee et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

Review Oncology

Balancing repair and tolerance of DNA damage caused by alkylating agents

Dragony Fu et al.

NATURE REVIEWS CANCER (2012)

Article Multidisciplinary Sciences

Non-ATG-initiated translation directed by microsatellite expansions

Tao Zu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2011)

Review Genetics & Heredity

Mechanisms of trinucleotide repeat instability during human development

Cynthia T. McMurray

NATURE REVIEWS GENETICS (2010)

Article Multidisciplinary Sciences

A simple ligand that selectively targets CUG trinucleotide repeats and inhibits MBNL protein binding

Jonathan F. Arambula et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)

Review Biotechnology & Applied Microbiology

Perspectives on NMR in drug discovery: a technique comes of age

Maurizio Pellecchia et al.

NATURE REVIEWS DRUG DISCOVERY (2008)

Review Multidisciplinary Sciences

Expandable DNA repeats and human disease

Sergei M. Mirkin

NATURE (2007)

Article Multidisciplinary Sciences

Reversal of RNA missplicing and myotonia after muscleblind overexpression in a mouse poly(CUG) model for myotonic dystrophy

Rahul N. Kanadia et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2006)

Review Biotechnology & Applied Microbiology

Chemical modifiers of unstable expanded simple sequence repeats: What goes up, could come down

Mario Gomes-Pereira et al.

MUTATION RESEARCH-FUNDAMENTAL AND MOLECULAR MECHANISMS OF MUTAGENESIS (2006)

Article Biochemistry & Molecular Biology

Chemotherapeutic deletion of CTG repeats in lymphoblast cells from DM1 patients

VI Hashem et al.

NUCLEIC ACIDS RESEARCH (2004)

Article Multidisciplinary Sciences

A muscleblind knockout model for myotonic dystrophy

RN Kanadia et al.

SCIENCE (2003)

Article Biochemistry & Molecular Biology

Muscleblind localizes to nuclear foci of aberrant RNA in myotonic dystrophy types 1 and 2

A Mankodi et al.

HUMAN MOLECULAR GENETICS (2001)