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Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.
Molecular therapy : the journal of the American Society of Gene Therapy 05, 2017 | Pubmed ID: 28389320
Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases.
Hematology/oncology clinics of North America 10, 2017 | Pubmed ID: 28895853
Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.
Science advances 12, 2017 | Pubmed ID: 29226242
Metallothioneins are neuroprotective agents in lysosomal storage disorders.
Annals of neurology 02, 2018 | Pubmed ID: 29369397
Gene Therapy as a Curative Option for β-Thalassemia.
The New England journal of medicine 04, 2018 | Pubmed ID: 29669229
Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis?
JAMA neurology 08, 2018 | Pubmed ID: 29913002
Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes.
Frontiers in immunology , 2018 | Pubmed ID: 29971065
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.
Blood advances 10, 2018 | Pubmed ID: 30282642
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Nature medicine 05, 2019 | Pubmed ID: 30911135
Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug release.
Biomaterials Jul, 2019 | Pubmed ID: 31026609
Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Gene Therapy Settings.
Human gene therapy 09, 2019 | Pubmed ID: 31037976
Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.
Molecular therapy : the journal of the American Society of Gene Therapy 07, 2019 | Pubmed ID: 31060789
A comprehensive single cell transcriptional landscape of human hematopoietic progenitors.
Nature communications 06, 2019 | Pubmed ID: 31160568
Gene-Based Approaches to Inherited Neurometabolic Diseases.
Human gene therapy 10, 2019 | Pubmed ID: 31397176
Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.
Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation Sep, 2019 | Pubmed ID: 31499213
Dana-Farber/Boston Children's Cancer and Blood Disorders Center
Harvard Medical School
Francisco J. Molina Estevez*,1,
Tyler D. Mathews*,1,
Alessandra Biffi1,2,
Marco Peviani1,2
1, Dana-Farber/Boston Children's Cancer and Blood Disorders Center,
2, Harvard Medical School
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