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Publications by Anna Villa
Analysis of Mutations From SCID and Omenn Syndrome Patients Reveals the Central Role of the Rag2 PHD Domain in Regulating V(D)J Recombination
Journal of Clinical Investigation
Medicine
Lentiviral Haemopoietic Stem/Progenitor Cell Gene Therapy for Treatment of Wiskott-Aldrich Syndrome: Interim Results of a Non-Randomised, Open-Label, Phase 1/2 Clinical Study
The Lancet Haematology
Hematology
447. Lentiviral Gene Therapy in a Preclinical Model of Omenn Syndrome
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Expanded Circulating Hematopoietic Stem/Progenitor Cells as Novel Cell Source for the Treatment of TCIRG1 Osteopetrosis
Haematologica
Hematology
Targeted NGS Platforms for Genetic Screening and Gene Discovery in Primary Immunodeficiencies
Frontiers in Immunology
Allergy
Immunology
As Little as Needed: The Extraordinary Case of a Mild Recessive Osteopetrosis Owing to a Novel Splicing Hypomorphic Mutation in theTCIRG1Gene
Journal of Bone and Mineral Research
Endocrinology
Orthopedics
Sports Medicine
Metabolism
Diabetes
RANK-dependent Autosomal Recessive Osteopetrosis: Characterization of Five New Cases With Novel Mutations
Journal of Bone and Mineral Research
Endocrinology
Orthopedics
Sports Medicine
Metabolism
Diabetes
One Disease, Many Genes: Implications for the Treatment of Osteopetroses
Frontiers in Endocrinology
Endocrinology
Metabolism
Diabetes
A Pre-Screening FISH-based Method to Detect CRISPR/Cas9 Off-Targets in Mouse Embryonic Stem Cells
Scientific Reports
Multidisciplinary
AIRE Deficiency in Thymus of 2 Patients With Omenn Syndrome
Journal of Clinical Investigation
Medicine
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