Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective.


Journal

Human gene therapy
ISSN: 1557-7422
Titre abrégé: Hum Gene Ther
Pays: United States
ID NLM: 9008950

Informations de publication

Date de publication:
10 2021
Historique:
pubmed: 9 9 2021
medline: 1 2 2022
entrez: 8 9 2021
Statut: ppublish

Résumé

Recent advances in genome editing tools, especially novel developments in the clustered regularly interspaced short palindromic repeats associated to Cas9 nucleases (CRISPR/Cas9)-derived editing machinery, have revolutionized not only basic science but, importantly, also the gene therapy field. Their flexibility and ability to introduce precise modifications in the genome to disrupt or correct genes or insert expression cassettes in safe harbors in the genome underline their potential applications as a medicine of the future to cure many genetic diseases. In this review, we give an overview of the recent progress made by French researchers in the field of therapeutic genome editing, while putting their work in the general context of advances made in the field. We focus on recent hematopoietic stem cell gene editing strategies for blood diseases affecting the red blood cells or blood coagulation as well as lysosomal storage diseases. We report on a genome editing-based therapy for muscular dystrophy and the potency of T cell gene editing to increase anticancer activity of chimeric antigen receptor T cells to combat cancer. We will also discuss technical obstacles and side effects such as unwanted editing activity that need to be surmounted on the way toward a clinical implementation of genome editing. We propose here improvements developed today, including by French researchers to overcome the editing-related genotoxicity and improve editing precision by the use of novel recombinant nuclease-based systems such as nickases, base editors, and prime editors. Finally, a solution is proposed to resolve the cellular toxicity induced by the systems employed for gene editing machinery delivery.

Identifiants

pubmed: 34494480
doi: 10.1089/hum.2021.191
doi:

Substances chimiques

Endonucleases EC 3.1.-

Types de publication

Journal Article Research Support, Non-U.S. Gov't Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

1059-1075

Auteurs

Mario Amendola (M)

Genethon, Evry, France.
Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.

Aurélie Bedel (A)

Bordeaux University, Bordeaux, France.
INSERM U1035, Biotherapy of Genetic Diseases, Inflammatory Disorders and Cancers, Bordeaux, France.
Biochemistry Laboratory, University Hospital Bordeaux, Bordeaux, France.

Ana Buj-Bello (A)

Genethon, Evry, France.
Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.

Mathieu Carrara (M)

Museum National d'Histoire Naturelle, Inserm U1154, CNRS UMR 7196, Sorbonne Universités, Paris, France.

Jean-Paul Concordet (JP)

Museum National d'Histoire Naturelle, Inserm U1154, CNRS UMR 7196, Sorbonne Universités, Paris, France.

Giacomo Frati (G)

Laboratory of Chromatin and Gene Regulation During Development, Imagine Institute, INSERM UMR1163, Paris, France.
Université de Paris, Paris, France.

David Gilot (D)

Inserm U1242, Université de Rennes, Centre de lutte contre le cancer Eugène Marquis, Rennes, France.

Carine Giovannangeli (C)

Museum National d'Histoire Naturelle, Inserm U1154, CNRS UMR 7196, Sorbonne Universités, Paris, France.

Alejandra Gutierrez-Guerrero (A)

CIRI-International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, Lyon, France.

Marine Laurent (M)

Genethon, Evry, France.
Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.

Annarita Miccio (A)

Laboratory of Chromatin and Gene Regulation During Development, Imagine Institute, INSERM UMR1163, Paris, France.
Université de Paris, Paris, France.

François Moreau-Gaudry (F)

Bordeaux University, Bordeaux, France.
INSERM U1035, Biotherapy of Genetic Diseases, Inflammatory Disorders and Cancers, Bordeaux, France.
Biochemistry Laboratory, University Hospital Bordeaux, Bordeaux, France.

Célia Sourd (C)

Genethon, Evry, France.
Université Paris-Saclay, Univ Evry, Inserm, Genethon, Integrare Research Unit UMR_S951, Evry, France.

Julien Valton (J)

Cellectis. SA, Paris, France.

Els Verhoeyen (E)

CIRI-International Center for Infectiology Research, Inserm, U1111, Université Claude Bernard Lyon 1, CNRS, UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon, Lyon, France.
Université Côte d'Azur, INSERM, C3M, Nice, France.

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Classifications MeSH