AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

[1]  G. Mentis,et al.  Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensory-motor circuit , 2021, Nature Neuroscience.

[2]  J. Lueck,et al.  Therapeutic promise of engineered nonsense suppressor tRNAs , 2021, Wiley interdisciplinary reviews. RNA.

[3]  Andrew Behrens,et al.  High-resolution quantitative profiling of tRNA abundance and modification status in eukaryotes by mim-tRNAseq , 2021, Molecular cell.

[4]  J. Eisengart,et al.  Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement , 2021, Biomolecules.

[5]  Johannes C. M. van der Loo,et al.  The clinical landscape for AAV gene therapies , 2021, Nature Reviews Drug Discovery.

[6]  S. Boye,et al.  Current Clinical Applications of In Vivo Gene Therapy with AAVs , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[7]  Mingyao Li,et al.  Adeno-Associated Virus-Induced Dorsal Root Ganglion Pathology. , 2020, Human gene therapy.

[8]  Gene-Wei Li,et al.  Growth-Optimized Aminoacyl-tRNA Synthetase Levels Prevent Maximal tRNA Charging. , 2020, Cell systems.

[9]  Ciaran M. Lee,et al.  AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9 , 2020, Molecular therapy : the journal of the American Society of Gene Therapy.

[10]  Alper Kucukural,et al.  DolphinNext: a distributed data processing platform for high throughput genomics , 2020, BMC Genomics.

[11]  F. Zhang,et al.  CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors , 2020, Cell.

[12]  E. Arbely,et al.  Translational read-through promotes aggregation and shapes stop codon identity , 2020, Nucleic acids research.

[13]  K. Rudolph,et al.  Smarcd3 is an epigenetic modulator of the metabolic landscape in pancreatic ductal adenocarcinoma , 2020, Nature Communications.

[14]  R. Samulski,et al.  Engineering adeno-associated virus vectors for gene therapy , 2020, Nature Reviews Genetics.

[15]  S. Mirarab,et al.  Sequence Analysis , 2020, Encyclopedia of Bioinformatics and Computational Biology.

[16]  Wei Yue,et al.  Semi-quantitative Determination of Protein Expression using Immunohistochemistry Staining and Analysis: An Integrated Protocol. , 2019, Bio-protocol.

[17]  P. Pinton,et al.  Translational readthrough of GLA nonsense mutations suggests dominant-negative effects exerted by the interaction of wild-type and missense variants , 2019, RNA biology.

[18]  Shuling Guo,et al.  Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations , 2019, Nucleic acid therapeutics.

[19]  P. Mali,et al.  immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy , 2019, Nature Biomedical Engineering.

[20]  L. Maquat,et al.  Quality and quantity control of gene expression by nonsense-mediated mRNA decay , 2019, Nature Reviews Molecular Cell Biology.

[21]  T. Inada,et al.  Translation arrest as a protein quality control system for aberrant translation of the 3′‐UTR in mammalian cells , 2019, FEBS letters.

[22]  G. Gao,et al.  Adeno-associated virus vector as a platform for gene therapy delivery , 2019, Nature Reviews Drug Discovery.

[23]  J. Lueck,et al.  Engineered transfer RNAs for suppression of premature termination codons , 2018, Nature Communications.

[24]  A. Pshezhetsky,et al.  Anatomical changes and pathophysiology of the brain in mucopolysaccharidosis disorders. , 2018, Molecular genetics and metabolism.

[25]  P. Zamore,et al.  Diseases caused by different mutations in the two alleles of a gene are treated in mice by Cas-9-induced allelic exchange. , 2018, Nature Biotechnology.

[26]  D. Bedwell,et al.  Identification of the amino acids inserted during suppression of CFTR nonsense mutations and determination of their functional consequences , 2017, Human molecular genetics.

[27]  Shoufang Gong,et al.  Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity. , 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

[28]  T. Pan,et al.  Determination of tRNA aminoacylation levels by high-throughput sequencing , 2017, Nucleic acids research.

[29]  Douglas R Martin,et al.  Direct Intracranial Injection of AAVrh8 Encoding Monkey β-N-Acetylhexosaminidase Causes Neurotoxicity in the Primate Brain. , 2017, Human gene therapy.

[30]  V. Gradinaru,et al.  Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems , 2017, Nature Neuroscience.

[31]  O. Mühlemann,et al.  Transcriptome-wide identification of NMD-targeted human mRNAs reveals extensive redundancy between SMG6- and SMG7-mediated degradation pathways , 2017, RNA.

[32]  A. Jacobson,et al.  Ataluren stimulates ribosomal selection of near-cognate tRNAs to promote nonsense suppression , 2016, Proceedings of the National Academy of Sciences.

[33]  Gaelen T. Hess,et al.  Translation readthrough mitigation , 2016, Nature.

[34]  D. Bedwell,et al.  Therapeutics based on stop codon readthrough. , 2014, Annual review of genomics and human genetics.

[35]  Björn Usadel,et al.  Trimmomatic: a flexible trimmer for Illumina sequence data , 2014, Bioinform..

[36]  A. T. van der Ploeg,et al.  Residual α-L-iduronidase activity in fibroblasts of mild to severe Mucopolysaccharidosis type I patients. , 2013, Molecular genetics and metabolism.

[37]  W. J. Ramsey,et al.  U6 promoter‐enhanced GlnUAG suppressor tRNA has higher suppression efficacy and can be stably expressed in 293 cells , 2013, The journal of gene medicine.

[38]  Anna M. McGeachy,et al.  The ribosome profiling strategy for monitoring translation in vivo by deep sequencing of ribosome-protected mRNA fragments , 2012, Nature Protocols.

[39]  L. Maquat,et al.  The Pioneer Round of Translation: Features and Functions , 2010, Cell.

[40]  Nicholas T. Ingolia,et al.  Genome-Wide Analysis in Vivo of Translation with Nucleotide Resolution Using Ribosome Profiling , 2009, Science.

[41]  A. Hinnebusch.  Translational regulation of GCN4 and the general amino acid control of yeast. , 2005, Annual review of microbiology.

[42]  Luc DesGroseillers,et al.  Mammalian Staufen1 Recruits Upf1 to Specific mRNA 3′UTRs so as to Elicit mRNA Decay , 2005, Cell.

[43]  D. Bedwell,et al.  Leaky termination at premature stop codons antagonizes nonsense-mediated mRNA decay in S. cerevisiae. , 2004, RNA.

[44]  R. Aebersold,et al.  A statistical model for identifying proteins by tandem mass spectrometry. , 2003, Analytical chemistry.

[45]  A. Davidoff,et al.  Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. , 2003, Blood.

[46]  Alexey I Nesvizhskii,et al.  Empirical statistical model to estimate the accuracy of peptide identifications made by MS/MS and database search. , 2002, Analytical chemistry.

[47]  R. Giegé,et al.  Identity of tRNA for yeast tyrosyl-tRNA synthetase: tyrosylation is more sensitive to identity nucleotides than to structural features. , 2000, Biochemistry.

[48]  R Giegé,et al.  Universal rules and idiosyncratic features in tRNA identity. , 1998, Nucleic acids research.

[49]  F. Jirik,et al.  Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene. , 1997, Human molecular genetics.

[50]  H. Beier,et al.  All human tRNATyr genes contain introns as a prerequisite for pseudouridine biosynthesis in the anticodon. , 1988, Nucleic acids research.

[51]  G. Temple,et al.  Construction of a functional human suppressor tRNA gene: an approach to gene therapy for β-thalassaemia , 1982, Nature.

[52]  G. Temple,et al.  Suppression of the nonsense mutation in homozygous β0 thalassaemia , 1979, Nature.

[53]  S. Cobb,et al.  Improved survival and reduced phenotypic severity following AAV9/MECP2 gene transfer to neonatal and juvenile male Mecp2 knockout mice. , 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

[54]  D. Bedwell,et al.  The designer aminoglycoside NB84 significantly reduces glycosaminoglycan accumulation associated with MPS I-H in the Idua-W392X mouse. , 2012, Molecular genetics and metabolism.

[55]  T. Schoeb,et al.  Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation. , 2010, Molecular genetics and metabolism.