AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

The clinical landscape for AAV gene therapies

(2021) Dmitry A. Kuzmin et al.   NATURE REVIEWS DRUG DISCOVERY

High-resolution quantitative profiling of tRNA abundance and modification status in eukaryotes by mim-tRNAseq

(2021) Andrew Behrens et al.   MOLECULAR CELL

Therapeutic promise of engineered nonsense suppressor tRNAs

(2021) Joseph J. Porter et al.   Wiley Interdisciplinary Reviews-RNA

Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement

(2021) Christiane S. Hampe et al.   Biomolecules

Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensorimotor circuit

(2021) Meaghan Van Alstyne et al.   NATURE NEUROSCIENCE

Engineering adeno-associated virus vectors for gene therapy

(2020) Chengwen Li et al.   NATURE REVIEWS GENETICS

Translational read-through promotes aggregation and shapes stop codon identity

(2020) Lior Kramarski et al.   NUCLEIC ACIDS RESEARCH

Stop codon context influences genome-wide stimulation of termination codon readthrough by aminoglycosides

(2020) Jamie R Wangen et al.   eLife

DolphinNext: a distributed data processing platform for high throughput genomics

(2020) Onur Yukselen et al.   BMC GENOMICS

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

(2020) Dan Wang et al.   CELL

AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

(2020) Ang Li et al.   MOLECULAR THERAPY

Growth-Optimized Aminoacyl-tRNA Synthetase Levels Prevent Maximal tRNA Charging

(2020) Darren J. Parker et al.   Cell Systems

Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology

(2020) Juliette Hordeaux et al.   HUMAN GENE THERAPY

Current Clinical Applications of In Vivo Gene Therapy with AAVs

(2020) Jerry R. Mendell et al.   MOLECULAR THERAPY

Engineered transfer RNAs for suppression of premature termination codons

(2019) John D. Lueck et al.   Nature Communications

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Translation arrest as a protein quality control system for aberrant translation of the 3′‐UTR in mammalian cells

(2019) Satoshi Hashimoto et al.   FEBS LETTERS

Quality and quantity control of gene expression by nonsense-mediated mRNA decay

(2019) Tatsuaki Kurosaki et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations

(2019) Lulu Huang et al.   Nucleic Acid Therapeutics

Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy

(2019) Ana M. Moreno et al.   Nature Biomedical Engineering

Translational readthrough of GLA nonsense mutations suggests dominant-negative effects exerted by the interaction of wild-type and missense variants

(2019) Silvia Lombardi et al.   RNA Biology

Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders

(2018) Brian W. Bigger et al.   MOLECULAR GENETICS AND METABOLISM

Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

(2018) Dan Wang et al.   NATURE BIOTECHNOLOGY

Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain

(2017) Diane Golebiowski et al.   HUMAN GENE THERAPY

Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences

(2017) Xiaojiao Xue et al.   HUMAN MOLECULAR GENETICS

Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

(2017) Jun Xie et al.   MOLECULAR THERAPY

Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems

(2017) Ken Y Chan et al.   NATURE NEUROSCIENCE

Determination of tRNA aminoacylation levels by high-throughput sequencing

(2017) Molly E. Evans et al.   NUCLEIC ACIDS RESEARCH

Translation readthrough mitigation

(2016) Joshua A. Arribere et al.   NATURE

Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression

(2016) Bijoyita Roy et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Transcriptome-wide identification of NMD-targeted human mRNAs reveals extensive redundancy between SMG6- and SMG7-mediated degradation pathways

(2016) Martino Colombo et al.   RNA

Therapeutics Based on Stop Codon Readthrough

(2014) Kim M. Keeling et al.   Annual Review of Genomics and Human Genetics

Trimmomatic: a flexible trimmer for Illumina sequence data

(2014) Anthony M. Bolger et al.   BIOINFORMATICS

U6 promoter-enhanced GlnUAG suppressor tRNA has higher suppression efficacy and can be stably expressed in 293 cells

(2013) Ramesh Koukuntla et al.   JOURNAL OF GENE MEDICINE

Residual α-l-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients

(2013) Esmee Oussoren et al.   MOLECULAR GENETICS AND METABOLISM

STAR: ultrafast universal RNA-seq aligner

(2012) Alexander Dobin et al.   BIOINFORMATICS

Improved Survival and Reduced Phenotypic Severity Following AAV9/MECP2 Gene Transfer to Neonatal and Juvenile Male Mecp2 Knockout Mice

(2012) Kamal KE Gadalla et al.   MOLECULAR THERAPY

The ribosome profiling strategy for monitoring translation in vivo by deep sequencing of ribosome-protected mRNA fragments

(2012) Nicholas T Ingolia et al.   Nature Protocols

The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse

(2011) Dan Wang et al.   MOLECULAR GENETICS AND METABOLISM

The Pioneer Round of Translation: Features and Functions

(2010) Lynne E. Maquat et al.   CELL

Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation

(2009) Dan Wang et al.   MOLECULAR GENETICS AND METABOLISM

Genome-Wide Analysis in Vivo of Translation with Nucleotide Resolution Using Ribosome Profiling

(2009) N. T. Ingolia et al.   SCIENCE