Helen de Boer

A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Salima Hacein‐Bey‐Abina, Sung‐Yun Pai, H. Bobby Gaspar et al. · New England Journal of Medicine · 2014 · 410 citations · Full text

Life Sciences, Immunology, Molecular Biology +13

miRNA-embedded shRNAs for Lineage-specific BCL11A Knockdown and Hemoglobin F Induction

Swaroopa Guda, Christian Brendel, Raffaele Renella et al. · Molecular Therapy · 2015 · 120 citations · Full text

Life Sciences, Genetics, Molecular Biology +16

Correction of Murine Rag2 Severe Combined Immunodeficiency by Lentiviral Gene Therapy Using a Codon-optimized RAG2 Therapeutic Transgene

Niek P. van Til, Helen de Boer, Nomusa Mashamba et al. · Molecular Therapy · 2012 · 70 citations · Full text

Life Sciences, Immunology, Molecular Genetics +16

Next Generation Sequencing Reveals Skewing of the T and B Cell Receptor Repertoires in Patients with Wiskott–Aldrich Syndrome

Amy E. Oâ€TMConnell, Stefano Volpi, Kerry Dobbs et al. · Frontiers in Immunology · 2014 · 45 citations · Full text

Life Sciences, Immunology, Molecular Biology +17

Lentiviral Hematopoietic Stem Cell Gene Therapy Corrects Murine Pompe Disease

Merel Stok, Helen de Boer, Marshall W. Huston et al. · Molecular Therapy — Methods & Clinical Development · 2020 · 36 citations · Full text

Life Sciences, Genetics, Molecular Genetics +14