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C

Céline J. Rocca

Genethon

H指数:14

被引数:953

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FAP-CAR-T cells reduce dystrophic muscle fibrosis, improving Adeno-Associated Virus gene transfer efficacy

FAP-CAR-T细胞减轻营养不良性肌肉纤维化，提高腺相关病毒基因转移效率

Molecular Therapy Methods & Clinical Development

IF4.7

2025-07-300

OAAI

Novel insights advancing the understanding of renal Fanconi syndrome in cystinosis

MOLECULAR GENETICS AND METABOLISM

IF3.5

2024-02-010

PRE

Upregulation of the Rab27a-Dependent Trafficking and Secretory Mechanisms Improves Lysosomal Transport, Alleviates Endoplasmic Reticulum Stress, and Reduces Lysosome Overload in Cystinosis

MOLECULAR AND CELLULAR BIOLOGY

IF2.7

2023-03-2046

OAAI

Interaction between galectin-3 and cystinosin uncovers a pathogenic role of inflammation in kidney involvement of cystinosis

KIDNEY INTERNATIONAL

IF12.6

2019-08-0126

OAAI

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency

SCIENTIFIC REPORTS

IF3.9

2018-05-295

OAAI

New interaction between galectin-3 and cystinosin reveals a role of inflammation in kidney pathogenesis in cystinosis

MOLECULAR GENETICS AND METABOLISM

IF3.5

2018-02-011

PREAI

Transplantation of wild-type mouse hematopoietic stem and progenitor cells ameliorates deficits in a mouse model of Friedreich's ataxia

SCIENCE TRANSLATIONAL MEDICINE

IF14.6

2017-10-2551

OAAI

Cystinosin, the small GTPase Rab11, and the Rab7 effector RILP regulate intracellular trafficking of the chaperone-mediated autophagy receptor LAMP2A

JOURNAL OF BIOLOGICAL CHEMISTRY

IF3.9

2017-06-0169

OAAI

Towards a phase I clinical trial for autologous hematopoietic stem cells transplantation in cystinosis

MOLECULAR GENETICS AND METABOLISM

IF3.5

2017-01-010

PREAI

Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model

MOLECULAR THERAPY

IF12

2016-05-010

OAAI

Towards a Phase I Clinical Trial for Cystinosis

MOLECULAR THERAPY

IF12

2016-05-012

OAAI

Kidney-Directed Gene Therapy for Murine Glycogen Storage Disease Type IA

MOLECULAR THERAPY

IF12

2016-05-010

OAAI

Hematopoietic Stem Cells Transplantation Can Normalize Thyroid Function in a Cystinosis Mouse Model

IF3.3

2016-01-2634

OAAI

Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE

IF4.7

2015-11-0531

OAAI

BRCA2 is needed for both repair and cell cycle arrest in mammalian cells exposed to S23906, an anticancer monofunctional DNA binder

IF3.4

2015-06-2611

OAAI

Pharmacology/Toxicology Studies for Gene-Modified Hematopoietic Stem Cell Transplantation for Cystinosis

MOLECULAR THERAPY

IF12

2015-05-010

OAAI

Treatment of Inherited Eye Defects By Systemic Hematopoietic Stem Cell Transplantation

MOLECULAR THERAPY

IF12

2015-05-010

OAAI

Impairment of chaperone-mediated autophagy leads to selective lysosomal degradation defects in the lysosomal storage disease cystinosis

分子伴侣介导的自噬功能受损导致溶酶体贮积症中选择性溶酶体降解缺陷

EMBO MOLECULAR MEDICINE

IF8.3

2015-01-1381

OAAI

Brief Reports: Lysosomal Cross-Correction by Hematopoietic Stem Cell-Derived Macrophages Via Tunneling Nanotubes

IF3.6

2014-12-18100

OAAI

Hematopoietic Stem Cell Gene Therapy for the Multisystemic Lysosomal Storage Disorder Cystinosis

MOLECULAR THERAPY

IF12

2013-02-0180

OAAI