Literature DB >> 27267267

Investor Outlook: Rising from the Ashes; GSK's European Approval of Strimvelis for ADA-SCID.

Joshua Schimmer1, Steven Breazzano1.   

Abstract

GlaxoSmithKline's (GSK) and partner San Raffaele Telethon Institute for Gene Therapy's recent positive European approval for Strimvelis for treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) represents the second EU-approved gene therapy and the first γ-retrovirus and first ex vivo gene therapy. In this article we discuss the significance and implications of this historic approval for the broader gene therapy field.

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Year:  2016        PMID: 27267267     DOI: 10.1089/humc.2016.29010.ind

Source DB:  PubMed          Journal:  Hum Gene Ther Clin Dev        ISSN: 2324-8637            Impact factor:   5.032


  7 in total

Review 1.  Stimulus-responsive viral vectors for controlled delivery of therapeutics.

Authors:  Mitchell J Brun; Eric J Gomez; Junghae Suh
Journal:  J Control Release       Date:  2017-08-24       Impact factor: 9.776

Review 2.  The Protective A673T Mutation of Amyloid Precursor Protein (APP) in Alzheimer's Disease.

Authors:  Qing Xia; XinYu Yang; JiaBin Shi; ZiJie Liu; YaHui Peng; WenJing Wang; BoWen Li; Yu Zhao; JiaYing Xiao; Lei Huang; DaYong Wang; Xu Gao
Journal:  Mol Neurobiol       Date:  2021-04-29       Impact factor: 5.590

3.  Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis.

Authors:  Lucia Monaco; Lucia Faccio
Journal:  EMBO Mol Med       Date:  2017-03       Impact factor: 12.137

4.  Toll-like receptor 2 promiscuity is responsible for the immunostimulatory activity of nucleic acid nanocarriers.

Authors:  Malvina Pizzuto; Monique Gangloff; Daniel Scherman; Nicholas J Gay; Virginie Escriou; Jean-Marie Ruysschaert; Caroline Lonez
Journal:  J Control Release       Date:  2016-12-28       Impact factor: 9.776

5.  Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.

Authors:  Marti Cabanes-Creus; Samantha L Ginn; Anais K Amaya; Sophia H Y Liao; Adrian Westhaus; Claus V Hallwirth; Patrick Wilmott; Jason Ward; Kimberley L Dilworth; Giorgia Santilli; Arkadiusz Rybicki; Hiroyuki Nakai; Adrian J Thrasher; Adrian C Filip; Ian E Alexander; Leszek Lisowski
Journal:  Mol Ther Methods Clin Dev       Date:  2018-11-01       Impact factor: 6.698

Review 6.  Engineering precision therapies: lessons and motivations from the clinic.

Authors:  Mingqi Xie; Mirta Viviani; Martin Fussenegger
Journal:  Synth Biol (Oxf)       Date:  2020-11-24

Review 7.  Clinical development of CAR T cells-challenges and opportunities in translating innovative treatment concepts.

Authors:  Jessica Hartmann; Martina Schüßler-Lenz; Attilio Bondanza; Christian J Buchholz
Journal:  EMBO Mol Med       Date:  2017-09       Impact factor: 12.137

  7 in total

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