Gene therapy on the move
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TLDR
This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival of gene Therapy as a powerful treatment option for the Correction of monogenic disorders.Abstract:
The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the occurrence of severe side effects in a few treated patients. The understanding of the molecular and cellular mechanisms leading to treatment- and/or vector-associated setbacks has resulted in the development of highly sophisticated gene transfer tools with improved safety and therapeutic efficacy. Employing these advanced tools, a series of Phase I/II trials were started in the past few years with excellent clinical results and no side effects reported so far. Moreover, highly efficient gene targeting strategies and site-directed gene editing technologies have been developed and applied clinically. With more than 1900 clinical trials to date, gene therapy has moved from a vision to clinical reality. This review focuses on the application of gene therapy for the correction of inherited diseases, the limitations and drawbacks encountered in some of the early clinical trials and the revival of gene therapy as a powerful treatment option for the correction of monogenic disorders.read more
Citations
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Therapeutic approaches for cardiac regeneration and repair
TL;DR: Advances in therapeutic approaches for cardiac repair and regeneration are discussed, including cell-based therapies as well as the use of secretory factors, such as microRNAs and exosomes, direct reprogramming strategies, and gene editing to control cardiac remodelling and redirect the adult heart to a regenerative state, and the future prospects of preclinical and clinical trials of heart regeneration.
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Teratozoospermia: spotlight on the main genetic actors in the human
Charles Coutton,Jessica Escoffier,Guillaume Martinez,Christophe Arnoult,Pierre F. Ray,Pierre F. Ray +5 more
TL;DR: Molecular studies of numerous unrelated patients with globozoospermia and large-headed spermatozoa confirmed that mutations in DPY19L2 and AURKC are mainly responsible for their respective pathological phenotype, strengthening the emerging point of view that MMAF may be a phenotypic variation of the classical forms of primary ciliary dyskinesia.
Journal ArticleDOI
Gene Therapy Leaves a Vicious Cycle.
Reena Goswami,Gayatri Subramanian,Liliya Silayeva,Isabelle Newkirk,Deborah M. Doctor,Karan Chawla,Saurabh Chattopadhyay,Dhyan Chandra,Nageswararao Chilukuri,Venkaiah Betapudi,Venkaiah Betapudi +10 more
TL;DR: The present review article delves into the popular viral vectors used in gene therapy, advances, challenges, and perspectives.
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Refining strategies to translate genome editing to the clinic
TL;DR: The advances made in the gene-editing field in recent years are discussed, and priorities that need to be addressed to expand therapeutic genome editing to further disease entities are specified.
Journal ArticleDOI
The approved gene therapy drugs worldwide: from 1998 to 2019
TL;DR: This review summarizes the gene therapy drugs approved worldwide from 1998 to 2019 in details, including names, indications, dates of approval, companies, vectors, the applied technologies and mechanisms of gene Therapy drugs, etc.
References
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LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.
Salima Hacein-Bey-Abina,C von Kalle,C von Kalle,Manfred Schmidt,Matthew P. McCormack,NM Wulffraat,Philippe Leboulch,Annick Lim,Cameron S. Osborne,R. Pawliuk,Estelle Morillon,R. Sorensen,A. Forster,Peter Fraser,Jeffrey I. Cohen,G de Saint Basile,Ian E. Alexander,Uwe Wintergerst,Thierry Frebourg,Alain Aurias,Dominique Stoppa-Lyonnet,Serge Romana,I. Radford-Weiss,Fabian Gross,Françoise Valensi,Eric Delabesse,Elizabeth Macintyre,F. Sigaux,Jean Soulier,L. E. Leiva,Manuela Wissler,Claudia Prinz,Terence H. Rabbitts,F. Le Deist,Alain Fischer,Marina Cavazzana-Calvo +35 more
TL;DR: Retrovirus vector insertion can trigger deregulated premalignant cell proliferation with unexpected frequency, most likely driven by retrovirus enhancer activity on the LMO2 gene promoter.
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