ORCID Profile
0000-0003-0582-540X
Current Organisations
Great Ormond Street Hospital For Children NHS Foundation Trust
,
University College London
Does something not look right? The information on this page has been harvested from data sources that may not be up to date. We continue to work with information providers to improve coverage and quality. To report an issue, use the Feedback Form.
Related Links
Publications
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
Publisher: Mary Ann Liebert Inc
Date: 2019
DOI: 10.1089/HUM.2018.098
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys
Publisher: Elsevier BV
Date: 12-2021
Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects
Publisher: Wiley
Date: 31-05-2017
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism
Publisher: American Association for the Advancement of Science (AAAS)
Date: 19-05-2021
DOI: 10.1126/SCITRANSLMED.AAW1564
Abstract: Viral vectors restore dopamine transporter function and ameliorate neuropathology in iPSC-derived neurons and a mouse model of infantile parkinsonism.
Delivering efficient liver-directed AAV-mediated gene therapy.
Publisher: Springer Science and Business Media LLC
Date: 12-01-2017
DOI: 10.1038/GT.2016.90
In vivo high-throughput profiling of CRISPR–Cpf1 activity
Publisher: Springer Science and Business Media LLC
Date: 19-12-2016
DOI: 10.1038/NMETH.4104
Abstract: CRISPR from Prevotella and Francisella 1 (Cpf1) is an effector endonuclease of the class 2 CRISPR-Cas (clustered regularly interspaced short palindromic repeats-CRISPR-associated proteins) gene editing system. We developed a method for evaluating Cpf1 activity, based on target sequence composition in mammalian cells, in a high-throughput manner. A library of >11,000 target sequence and guide RNA pairs was delivered into human cells using lentiviral vectors. Subsequent delivery of Cpf1 into this cell library induced insertions and deletions (indels) at the integrated synthetic target sequences, which allowed en masse evaluation of Cpf1 activity by using deep sequencing. With this approach, we determined protospacer-adjacent motif sequences of two Cpf1 nucleases, one from Acidaminococcus sp. BV3L6 (hereafter referred to as AsCpf1) and the other from Lachnospiraceae bacterium ND2006 (hereafter referred to as LbCpf1). We also defined target-sequence-dependent activity profiles of AsCpf1, which enabled the development of a web tool that predicts the indel frequencies for given target sequences (big.hanyang.ac.kr/cindel). Both the Cpf1 characterization profile and the in vivo high-throughput evaluation method will greatly facilitate Cpf1-based genome editing.
Related Organisations
Great Ormond Street Hospital For Children NHS Trust
Location: United Kingdom of Great Britain and Northern Ireland
Great Ormond Street Hospital For Children NHS Foundation Trust
Location: United Kingdom of Great Britain and Northern Ireland
University College London
Location: United Kingdom of Great Britain and Northern Ireland
Related Funding Activities
No related grants have been discovered for Julien Baruteau.