Literature DB >> 29575374

Gene therapy clinical trials worldwide to 2017: An update.

Samantha L Ginn1, Anais K Amaya1, Ian E Alexander1,2, Michael Edelstein3, Mohammad R Abedi4.   

Abstract

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have been approved worldwide. Our database brings together global information on gene therapy clinical activity from trial databases, official agency sources, published literature, conference presentations and posters kindly provided to us by individual investigators or trial sponsors. This review presents our analysis of clinical trials that, to the best of our knowledge, have been or are being performed worldwide. As of our November 2017 update, we have entries on 2597 trials undertaken in 38 countries. We have analysed the geographical distribution of trials, the disease indications (or other reasons) for trials, the proportions to which different vector types are used, and the genes that have been transferred. Details of the analyses presented, and our searchable database are available via The Journal of Gene Medicine Gene Therapy Clinical Trials Worldwide website at: http://www.wiley.co.uk/genmed/clinical. We also provide an overview of the progress being made in gene therapy clinical trials around the world, and discuss key trends since the previous review, namely the use of chimeric antigen receptor T cells for the treatment of cancer and advancements in genome editing technologies, which have the potential to transform the field moving forward.
Copyright © 2018 John Wiley & Sons, Ltd.

Entities:  

Keywords:  The Journal of Gene Medicine; clinical trials; database; gene therapy; worldwide

Mesh:

Year:  2018        PMID: 29575374     DOI: 10.1002/jgm.3015

Source DB:  PubMed          Journal:  J Gene Med        ISSN: 1099-498X            Impact factor:   4.565


  169 in total

1.  Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity.

Authors:  Mickaël Guilbaud; Marie Devaux; Celia Couzinié; Johanne Le Duff; Alice Toromanoff; Céline Vandamme; Nicolas Jaulin; Gwladys Gernoux; Thibaut Larcher; Philippe Moullier; Caroline Le Guiner; Oumeya Adjali
Journal:  Hum Gene Ther       Date:  2019-04-16       Impact factor: 5.695

Review 2.  Challenges of gene delivery to the central nervous system and the growing use of biomaterial vectors.

Authors:  Devan L Puhl; Anthony R D'Amato; Ryan J Gilbert
Journal:  Brain Res Bull       Date:  2019-06-05       Impact factor: 4.077

3.  Biopharmaceutical benchmarks 2018.

Authors:  Gary Walsh
Journal:  Nat Biotechnol       Date:  2018-12-06       Impact factor: 54.908

4.  An integrative method to predict signalling perturbations for cellular transitions.

Authors:  Gaia Zaffaroni; Satoshi Okawa; Manuel Morales-Ruiz; Antonio Del Sol
Journal:  Nucleic Acids Res       Date:  2019-07-09       Impact factor: 16.971

5.  An Engineered Galactosylceramidase Construct Improves AAV Gene Therapy for Krabbe Disease in Twitcher Mice.

Authors:  Xiufang Pan; Scott A Sands; Yongping Yue; Keqing Zhang; Steven M LeVine; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2019-07-18       Impact factor: 5.695

Review 6.  BMP gene delivery for skeletal tissue regeneration.

Authors:  Maxim Bez; Gadi Pelled; Dan Gazit
Journal:  Bone       Date:  2020-05-21       Impact factor: 4.398

7.  Polyamidoamine Dendrimer Grafted with an Acid-Responsive Charge-Reversal Layer for Improved Gene Delivery.

Authors:  Juan Wang; Remy C Cooper; Hu Yang
Journal:  Biomacromolecules       Date:  2020-09-08       Impact factor: 6.988

Review 8.  A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs).

Authors:  Gearóid P Tuohy; Roly Megaw
Journal:  Biomolecules       Date:  2021-05-19

9.  Engineered materials for in vivo delivery of genome-editing machinery.

Authors:  Sheng Tong; Buhle Moyo; Ciaran M Lee; Kam Leong; Gang Bao
Journal:  Nat Rev Mater       Date:  2019-10-04       Impact factor: 66.308

Review 10.  Stem cell-based therapy for hirschsprung disease, do we have the guts to treat?

Authors:  Ali Fouad Alhawaj
Journal:  Gene Ther       Date:  2021-06-14       Impact factor: 5.250

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