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

Molecular Therapy — Methods & Clinical Development(2020)10.1016/j.omtm.2020.04.018Source: DataRank Database

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich’s Ataxia is a research paper published in Molecular Therapy — Methods & Clinical Development (2020). On theSindex it has a DataRank of 0. It has been cited 36 times.

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Open Access36 citations

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datarank_citation_only_1hop_v6· scope data_onlyMethodology

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Enrichment:Funding (9 grants)OA: goldTopics (34)IDs (PubMed, PMC)Annotations (2)SDGs (1)

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Findable (1/2)

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Accessible (1/2)

Open Access

Interoperable (0/2)

Reusable (0/3)

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Authors (8)

Joseph Rainaldi ORCID,Jay Sharma ORCID,Yanmeng Shi ORCID,Joseph H. Haquang,Jens Luebeck ORCID

Live enrichment

PubMed:32462051 PMC:PMC7240056

Cystinosis Research Foundation

Grant: EDUC2-08388

California Institute of Regenerative Medicine

Grant: CLIN-09230

National Institutes of Health

Grant: R01-NS108965

National Institutes of Health

Grant: RO1-DK090058

NIDDK NIH HHS

Grant: R01 DK090058

NINDS NIH HHS

Grant: R01 NS108965

National Institutes of Health

Grant: 5R01NS108965-04

Hematopoietic Stem Cell Gene Therapy for Friedreich's ataxia

National Institutes of Health

Grant: 2R01DK090058-06

Lentiviral-transduced hematopoictic stem cell transplantation for cystinosis

Friedreich's Ataxia Research Alliance

goldLicense: cc-by-nc-nd

publisherhttp://www.cell.com/article/S2329050120300784/pdf

publisherhttps://api.elsevier.com/content/article/PII:S2329050120300784?httpAccept=text/xml

publisherhttps://api.elsevier.com/content/article/PII:S2329050120300784?httpAccept=text/plain

repositoryhttps://doaj.org/article/363e1c35035d4f959e428be557dad5a2

repositoryhttps://www.ncbi.nlm.nih.gov/pmc/articles/7240056

Fields of Study

0301 basic medicine03 medical and health sciences0303 health sciences

Keywords

Hematopoietic stem cellsp53Neurodegenerative disorderFriedreich's AtaxiaEx Vivo Gene TherapyGene EditingFrataxinCrispr/cas9Mitochondrial DisorderMedical BiotechnologyNeurodegenerativeQH426-470Regenerative MedicineArticleRare DiseasesClinical ResearchStem Cell Research - Nonembryonic - HumanGenetics2.1 Biological and endogenous factorsAetiologyPediatricTransplantationBiomedical and Clinical Sciences5.2 Cellular and gene therapiesQH573-671NeurosciencesStem Cell ResearchNeurologicalDevelopment of treatments and therapeutic interventionsCytologyBiotechnology

Sustainable Development Goals

SDG 3: 3. Good health

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

FAIR Checklist

Authors (8)

Funding (9 grants)

Open Access

Topics

Annotations (Europe PMC)