Alessandra Biffi

Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.

Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases.

Intracerebroventricular delivery of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells.

Metallothioneins are neuroprotective agents in lysosomal storage disorders.

Gene Therapy as a Curative Option for β-Thalassemia.

Metachromatic Leukodystrophy: Too Frequent (Mis)Diagnosis?

Prostaglandin E2 Stimulates the Expansion of Regulatory Hematopoietic Stem and Progenitor Cells in Type 1 Diabetes.

Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.

Highly efficient therapeutic gene editing of human hematopoietic stem cells.

Biodegradable polymeric nanoparticles administered in the cerebrospinal fluid: Brain biodistribution, preferential internalization in microglia and implications for cell-selective drug release.

Assessing the Impact of Cyclosporin A on Lentiviral Transduction and Preservation of Human Hematopoietic Stem Cells in Clinically Relevant Gene Therapy Settings.

Targeting a Pre-existing Anti-transgene T Cell Response for Effective Gene Therapy of MPS-I in the Mouse Model of the Disease.

A comprehensive single cell transcriptional landscape of human hematopoietic progenitors.

Gene-Based Approaches to Inherited Neurometabolic Diseases.

Hematopoietic Stem Cell Transplantation to Treat Leukodystrophies: Clinical Practice Guidelines from the Hunter's Hope Leukodystrophy Care Network.