A Thrasher

Long-term efficacy and safety results of betibeglogene autotemcel gene therapy for transfusion-dependent β-thalassemia

J. Kwiatkowski, Alexis A. Thompson, Jennifer Schneiderman, Isabelle Thuret et autres

us, fr, de, gr, th, gb, au, it (code pays fourni par la source)

Immunodeficiency, autoimmunity, and increased risk of B cell malignancy in humans with TRAF3 mutations

William Rae, John M. Sowerby, Dorit Verhoeven, Mariam Youssef et autres

gb, nl, ca, us, Soudan du Sud, vn, mx (code pays fourni par la source)

Impact of HIV on postpartum hemorrhage in South Africa

A Thrasher, Motshedisi Sebitloane, Nuriya Robinson

us, Afrique du Sud (code pays fourni par la source)

Severe autoinflammatory disease caused by mutation in a gene controlling actin cytoskeletal dynamics and cure with allogeneic haematopoetic stem cell transplantation

Ariane Standing, Dessislava Malinova, Julien Record, Dale Moulding et autres

gb (code pays fourni par la source)

Site-specific gene editing of COL7A1 restores type VII collagen in RDEB iPSCs

Anastasia Petrova, Christos Georgiadis, A Thrasher, W. Di et autres

Correction of haemoglobin levels in a humanized mouse model of thalassemia after fetal gene therapy

Stavros Loukogeorgakis, Sindhu Subramaniam, Michail Antoniou, Nahla Bakhamis et autres

Functional human epidermal equivalent derived from induced pluripotent stem cells as a model to study genetic correction in Epidermolysis bullosa

Anastasia Petrova, Weihao Di, Duško Ilić, Theodora M. Mauro et autres

Production of gene engineered epithelia sheets for first-in-man use

W. Di, Sulung Rahmawan Wira Ghani, David Almarza, Jemima E. Mellerio et autres

Human artificial chromosomes maintained in human embryonic stem and neuronal cells

Daniela Moralli, Mohammad A. Mandegar, Suhail Khoja, Sally A. Cowley et autres

Prevention of silencing by UCOE deletion constructs

Walther Haenseler, Christian Brendel, G Santili, Williams James et autres

Viral expression of GM-CSF: neuroinflammatory response and effects on regeneration

C Acosta-Saltos, Roman Gonitel, Seetal Chavda, Alejandro Acosta‐Saltos et autres

Lentivirus mediated protection against HIV-1 using a human TRIM21-Cyclophilin A fusion protein

Eugene Y. Chan, Torsten Schaller, A Thrasher, Greg J. Towers et autres