In Vivo RNA Delivery to Hematopoietic Stem and Progenitor Cells via Targeted Lipid Nanoparticles

Lipid nanoparticle-mediated silencing of osteogenic suppressor GNAS leads to osteogenic differentiation of mesenchymal stem cells in vivo

(2022) Genc Basha et al.   MOLECULAR THERAPY

Ensuring a future for gene therapy for rare diseases

(2022) Alessandro Aiuti et al.   NATURE MEDICINE

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice

(2021) Chang Li et al.   Blood Advances

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

(2021) Donald B. Kohn et al.   NEW ENGLAND JOURNAL OF MEDICINE

Base editing of haematopoietic stem cells rescues sickle cell disease in mice

(2021) Gregory A. Newby et al.   NATURE

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

(2021) Bernhard Gentner et al.   NEW ENGLAND JOURNAL OF MEDICINE

Mast Cells Are Identified in the Lung Parenchyma of Wild Mice, Which Can Be Recapitulated in Naturalized Laboratory Mice

(2021) Yu-Wen Yeh et al.   Frontiers in Immunology

Anti-human CD117 CAR T-cells efficiently eliminate healthy and malignant CD117-expressing hematopoietic cells

(2020) Renier Myburgh et al.   LEUKEMIA

In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model

(2020) Chang Li et al.   MOLECULAR THERAPY

Nanoparticle-encapsulated siRNAs for gene silencing in the haematopoietic stem-cell niche

(2020) Marvin Krohn-Grimberghe et al.   Nature Biomedical Engineering

Gene therapy using haematopoietic stem and progenitor cells

(2020) Giuliana Ferrari et al.   NATURE REVIEWS GENETICS

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

(2020) Francesca Tucci et al.   MOLECULAR THERAPY

Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation

(2019) Agnieszka Czechowicz et al.   Nature Communications

Search-and-replace genome editing without double-strand breaks or donor DNA

(2019) Andrew V. Anzalone et al.   NATURE

In vivo hematopoietic stem cell gene therapy ameliorates murine thalassemia intermedia

(2018) Hongjie Wang et al.   JOURNAL OF CLINICAL INVESTIGATION

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

(2018) Cory D. Sago et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy

(2016) Akanksha Chhabra et al.   Science Translational Medicine

Dynamic equilibrium of heterogeneous and interconvertible multipotent hematopoietic cell subsets

(2014) Wendy Weston et al.   Scientific Reports

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

Complications of autologous hematopoietic stem cell transplantation for patients with autoimmune diseases

(2012) Thomas Daikeler et al.   PEDIATRIC RESEARCH

Stem Cell Factor Receptor/c-Kit: From Basic Science to Clinical Implications

(2012) Johan Lennartsson et al.   PHYSIOLOGICAL REVIEWS

A robust and high-throughput Cre reporting and characterization system for the whole mouse brain

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE