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Stéphanie Cherqui

university of california

H指数:33

被引数:3,683

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Comprehensive Ocular Characteristics in Cystinosis after Hematopoietic Stem-Cell Gene Therapy over 24 Months

24个月内造血干细胞基因治疗后胱氨酸病的全面眼部特征

American Journal of Ophthalmology

IF4.2

2026-02-090

PRE

Microgliopathy as a primary mediator of neuronal death in models of Friedreich’s Ataxia

小胶质细胞病作为弗里德赖希共济失调模型中神经元死亡的主要介导因素

Nature Communications

IF15.7

2025-11-290

OAAI

Transplantation of Wild-Type Hematopoietic Stem and Progenitor Cells Improves Disease Phenotypes in a Mucopolysaccharidosis IIIC Mouse Model

CELL TRANSPLANTATION

IF3.2

2025-01-010

OAAI

Targeted gene therapy for rare genetic kidney diseases

KIDNEY INTERNATIONAL

IF12.6

2024-12-010

OAAI

Reconstitution of Rab11-FIP4 Expression Rescues Cellular Homeostasis in Cystinosis

MOLECULAR AND CELLULAR BIOLOGY

IF2.7

2024-10-220

OAAI

Novel insights advancing the understanding of renal Fanconi syndrome in cystinosis

MOLECULAR GENETICS AND METABOLISM

IF3.5

2024-02-010

PRE

Y Gene modified hematopoietic stem cell transplantation for mucopolysaccharidosis type IIIC

MOLECULAR GENETICS AND METABOLISM

IF3.5

2024-02-010

PRE

Rescue of Alzheimer's disease phenotype in a mouse model by transplantation of wild-type hematopoietic stem and progenitor cells

IF6.9

2023-08-0113

OAAI

Intralysosomal cystine accumulation in mice lacking cystinosin, the protein defective in cystinosis

MOLECULAR AND CELLULAR BIOLOGY

IF2.7

2023-03-27148

OAAI

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

Hematopoietic stem cell gene therapy for mucopolysaccharidosis type IIIC

MOLECULAR GENETICS AND METABOLISM

IF3.5

2023-02-010

PRE

Phase 1/2 clinical trial of autologous hematopoietic stem and progenitor cell (HSPC) gene therapy for cystinosis

MOLECULAR GENETICS AND METABOLISM

IF3.5

2023-02-010

PRE

Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial

MOLECULAR GENETICS AND METABOLISM

IF3.5

2022-02-010

PRE

High-resolution cellular and molecular follow up of lysosomal disease patients treated with hematopoietic stem cell lentiviral gene therapy

MOLECULAR GENETICS AND METABOLISM

IF3.5

2022-02-010

PRE

Deficiency of the sedoheptulose kinase (Shpk) does not alter the ability of hematopoietic stem cells to rescue cystinosis in the mouse model

MOLECULAR GENETICS AND METABOLISM

IF3.5

2021-12-014

OAAI

Hematopoietic Stem Cell Gene Therapy for Cystinosis: From Bench-to-Bedside

IF5.2

2021-11-2314

OAAI

DYNC1LI2 regulates localization of the chaperone-mediated autophagy receptor LAMP2A and improves cellular homeostasis in cystinosis

IF14.3

2021-10-1312

OAAI

Hematopoietic stem cell gene therapy for cystinosis: Updated results from a phase I/II clinical trial

MOLECULAR GENETICS AND METABOLISM

IF3.5

2021-02-011

PREAI

Macrophage polarization impacts tunneling nanotube formation and intercellular organelle trafficking

SCIENTIFIC REPORTS

IF3.9

2019-10-1023

OAAI

Hematopoietic Stem Cell Gene Therapy for Brain Metastases Using Myeloid Cell-Specific Gene Promoters

JNCI-JOURNAL OF THE NATIONAL CANCER INSTITUTE

IF7.2

2019-09-1017

OAAI