In vivo hematopoietic stem cell modification by mRNA delivery

CAR T cells produced in vivo to treat cardiac injury

(2022) Joel G. Rurik et al.   SCIENCE

Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies

(2021) Laura Breda et al.   MOLECULAR THERAPY

Highly efficient CD4+ T cell targeting and genetic recombination using engineered CD4+ cell-homing mRNA-LNPs

(2021) István Tombácz et al.   MOLECULAR THERAPY

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

(2021) Gregory A. Newby et al.   NATURE

Selective targeting of nanomedicine to inflamed cerebral vasculature to enhance the blood–brain barrier

(2020) Oscar A. Marcos-Contreras et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lipid nanoparticles for nucleic acid delivery: Current perspectives

(2020) Eleni Samaridou et al.   ADVANCED DRUG DELIVERY REVIEWS

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

Low Exposure Busulfan Conditioning to Achieve Sufficient Multi-lineage Chimerism in Patients with Severe Combined Immunodeficiency

(2019) Christopher C. Dvorak et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

A Facile Method for the Removal of dsRNA Contaminant from In Vitro-Transcribed mRNA

(2019) Markus Baiersdörfer et al.   Molecular Therapy-Nucleic Acids

MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

(2018) Ruchi Jain et al.   Nucleic Acid Therapeutics

PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake

(2018) Hamideh Parhiz et al.   JOURNAL OF CONTROLLED RELEASE

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers

(2016) L. Breda et al.   BLOOD

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

(2016) Marina Cavazzana et al.   HUMAN GENE THERAPY

mRNA: Fulfilling the Promise of Gene Therapy

(2015) Drew Weissman et al.   MOLECULAR THERAPY

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

The human stem cell hierarchy is defined by a functional dependence on Mcl-1 for self-renewal capacity

(2010) C. J. V. Campbell et al.   BLOOD

Quantitatively different red cell/nucleated cell chimerism in patients with long-term, persistent hematopoietic mixed chimerism after bone marrow transplantation for thalassemia major or sickle cell disease

(2010) M. Andreani et al.   HAEMATOLOGICA

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Prospective isolation and molecular characterization of hematopoietic stem cells with durable self-renewal potential

(2009) D. G. Kent et al.   BLOOD

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

(2009) Linda Madisen et al.   NATURE NEUROSCIENCE

Endocrinological late complications after hematopoietic SCT in children

(2008) A Cohen et al.   BONE MARROW TRANSPLANTATION

Regulation of Hematopoietic Stem Cells by the Steel Factor/KIT Signaling Pathway

(2008) D. Kent et al.   CLINICAL CANCER RESEARCH

In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of  -thalassemia

(2008) A. Miccio et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA