A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia-Reference-Cited by-同舟云学术

A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia

Published:2021-10-29 Issue:44 Volume:7 Page:
ISSN:2375-2548
Container-title:Science Advances
language:en
Short-container-title:Sci. Adv.

Author:

Zhukouskaya Volha V.¹²³⁴^ORCID,Jauze Louisa¹²⁵^ORCID,Charles Séverine¹²,Leborgne Christian¹²^ORCID,Hilliquin Stéphane³⁶,Sadoine Jérémy³,Slimani Lotfi³,Baroukh Brigitte³,van Wittenberghe Laetitia¹,Danièle Natalie¹^ORCID,Rajas Fabienne⁵,Linglart Agnès⁴^ORCID,Mingozzi Federico¹²^ORCID,Chaussain Catherine³⁴⁷^ORCID,Bardet Claire³^ORCID,Ronzitti Giuseppe¹²^ORCID

Affiliation:

1. Genethon, 91000 Evry, France.

2. Université Paris-Saclay, Univ Evry, Inserm, Genethon, INTEGRARE Research Unit UMR_S951, 91000 Evry, France.

3. Université de Paris, Institut des maladies musculo-squelettiques, Laboratory Orofacial Pathologies, Imaging and Biotherapies URP2496 and FHU-DDS-Net, Dental School, and Plateforme d’Imagerie du Vivant (PIV), Montrouge, France.

4. Paris-Saclay University, INSERM U1185, AP-HP, DMU SEA, Endocrinology and Diabetes for Children, Reference Center for Rare Diseases of the Calcium and Phosphate Metabolism, OSCAR filière, EndoRare, and BOND ERN, Bicêtre Hospital, Le Kremlin-Bicêtre, France.

5. Institut National de la Santé et de la Recherche Médicale, U1213, Lyon F-69008, France.

6. AP-HP, Department of Rheumatology, Cochin Hospital, Université de Paris, Paris, France.

7. AP-HP, Reference Center for Rare Disorders of the Calcium and Phosphate Metabolism, Dental Medicine Department, Bretonneau Hospital, GHN-Université de Paris, Paris 75018, France.

Abstract

A new therapeutic approach for the treatment of a skeletal disorder with AAVs bypasses bone resistance to gene transfer.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference50 articles.

1. Clinical practice recommendations for the diagnosis and management of X-linked hypophosphataemia

2. FGF-23 Is a Potent Regulator of Vitamin D Metabolism and Phosphate Homeostasis

3. Therapeutic management of hypophosphatemic rickets from infancy to adulthood

4. FGF23 and its role in X-linked hypophosphatemia-related morbidity

5. Rickets

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1. The sacroiliac joint: An original and highly sensitive tool to highlight altered bone phenotype in murine models of skeletal disorders;Bone;2024-01

2. Blocking FGF23 signaling improves the growth plate of mice with X-linked hypophosphatemia;Journal of Endocrinology;2023-07-12

3. Current and Future Therapeutical Aspects of X-Linked Hypophosphatemia in Children With Special Attention to Klotho/Fibroblast Growth Factor 23 System;Journal of Clinical Medicine Research;2022-10