Journal of Stem Cell Research & Therapy

Humanized mice are precious tools for evaluation of hematopoietic gene therapies and preclinical modeling to move towards a clinical trial

Engineering precision therapies: lessons and motivations from the clinic

Genome writing: current progress and related applications

Gene therapy for X-linked severe combined immunodeficiency: historical outcomes and current status

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

Next generation sequencing in newborn screening in the United Kingdom National Health Service

Advances and highlights in primary immunodeficiencies in 2017

Thymus autonomy as a prelude to leukemia

Gene therapy using haematopoietic stem and progenitor cells

T cell acute lymphoblastic leukemia as a consequence of thymus autonomy

Gene therapy and genome editing for primary immunodeficiency diseases

CRISPR/Cas9-modified hematopoietic stem cells—present and future perspectives for stem cell transplantation

From stem cells to functional lymphocytes: cell differentiation and gene therapy implementation for RAG-SCID

Illuminating the genome-wide activity of genome editors for safe and effective therapeutics

Defining B cell tolerance checkpoints and their modulation by immunotherapy

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott-Aldrich Syndrome

 Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's

Self-renewal of double-negative 3 early thymocytes enables thymus autonomy but compromises the ?-selection checkpoint

p53 and Cell Fate: Sensitizing Head and Neck Cancer Stem Cells to Chemotherapy