More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

The experience of the past 3 years, since the first case of leukemia was reported in a child
cured of X-linked severe combined immunodeficiency (X-SCID) by gene therapy, indicates …

Deciphering how neural circuits are anatomically organized with regard to input and output
is instrumental in understanding how the brain processes information. For example, locus …

Fluorescent calcium sensors are widely used to image neural activity. Using structure-based
mutagenesis and neuron-based screening, we developed a family of ultrasensitive protein …

Chimeric antigen receptor (CAR) T-cell immunotherapy has revolutionized the treatment of
refractory leukemias and lymphomas, but is associated with significant toxicities, namely …

MDA5 is a cytosolic sensor of double-stranded RNA (ds) RNA including viral byproducts and
intermediates. We studied a child with life-threatening, recurrent respiratory tract infections …

Viruses have evolved specialized molecular mechanisms to transfer their genome efficiently
into host cells. Viruses can be repurposed into viral vectors to achieve controlled gene …

Murine leukemia virus (MLV)-derived vectors are widely used for hematopoietic stem cell
(HSC) gene transfer, but lentiviral vectors such as the simian immunodeficiency virus (SIV) …

Retroviral vector-mediated gene transfer has been central to the development of gene
therapy. Retroviruses have several distinct advantages over other vectors, especially when …

β-thalassemias are the most common single gene disorders and are potentially amenable to
gene therapy. However, retroviral vectors carrying the human β-globin cassette have been …