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List of works by Celine J Rocca

A southern blot protocol to detect chimeric nuclease-mediated gene repair.

scientific article published on January 2014

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

scientific article

Brief reports: Lysosomal cross-correction by hematopoietic stem cell-derived macrophages via tunneling nanotubes

scientific article

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia

scientific article published on 03 May 2020

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

scientific article published on 2 May 2017

Gene Transfer to Mouse Kidney In Vivo

scientific article published on 01 January 2019

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

scientific article published on 26 January 2016

Hematopoietic stem cell gene therapy for the multisystemic lysosomal storage disorder cystinosis

scientific article published on 23 October 2012

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

scientific article

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

scientific article published on 6 March 2019

Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.

scientific article

Potential use of stem cells as a therapy for cystinosis

scientific article published on 22 May 2018

The NER proteins XPC and CSB, but not ERCC1, regulate the sensitivity to the novel DNA binder S23906: implications for recognition and repair of antitumor alkylators.

scientific article

Trabectedin and Its C Subunit Modified Analogue PM01183 Attenuate Nucleotide Excision Repair and Show Activity toward Platinum-Resistant Cells ⬇️

scientific article published on May 27, 2011

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

scientific article published in October 2017

Treatment of Inherited Eye Defects by Systemic Hematopoietic Stem Cell Transplantation

scientific article

Upregulation of the Rab27a-dependent trafficking and secretory mechanisms improves lysosomal transport, alleviates endoplasmic reticulum stress, and reduces lysosome overload in cystinosis

scientific article

rAAV9 combined with renal vein injection is optimal for kidney-targeted gene delivery: conclusion of a comparative study

scientific article published on May 2014

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