Alessandra Biffi

Dana-Farber/Boston Children's Cancer and Blood Disorders Center

Affiliated withDana-Farber/Boston Children's Cancer and Blood Disorders Center Harvard Medical School

Research Area

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JoVE Journal Publications

ArticleTotal : 1	Year
Publication title Cited by 8	2019

Other Publications

Article	Year
Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 28389320	2017
Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases. Hematology/oncology clinics of North America\| PubMed ID: 28895853	2017
Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells. Science advances\| PubMed ID: 29226242	2017
Metallothioneins are neuroprotective agents in lysosomal storage disorders. Annals of neurology\| PubMed ID: 29369397	2018
Gene Therapy as a Curative Option for β-Thalassemia. The New England journal of medicine\| PubMed ID: 29669229	2018
Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis? JAMA neurology\| PubMed ID: 29913002	2018
Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes. Frontiers in immunology\| PubMed ID: 29971065	2018
Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients. Blood advances\| PubMed ID: 30282642	2018
Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nature medicine\| PubMed ID: 30911135	2019
Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug relea... Biomaterials\| PubMed ID: 31026609	2019
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\| PubMed ID: 31037976	2019
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\| PubMed ID: 31060789	2019
A comprehensive single cell transcriptional landscape of human hematopoietic progenitors. Nature communications\| PubMed ID: 31160568	2019
Gene-Based Approaches to Inherited Neurometabolic Diseases. Human gene therapy\| PubMed ID: 31397176	2019
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\| PubMed ID: 31499213	2019