Microglia were first recognized as a distinct cell population in the CNS one century ago. For
a long time, they were primarily considered to be phagocytes responsible for removing …

Lysosomal storage diseases (LSDs) are a group of over 70 diseases that are characterized
by lysosomal dysfunction, most of which are inherited as autosomal recessive traits. These …

Background Effective treatment for metachromatic leukodystrophy (MLD) remains a
substantial unmet medical need. In this study we investigated the safety and efficacy of …

BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

Viral vectors provide an efficient means for modification of eukaryotic cells, and their use is
now commonplace in academic laboratories and industry for both research and clinical …

Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

Background Allogeneic hematopoietic stem-cell transplantation is the standard of care for
Hurler syndrome (mucopolysaccharidosis type I, Hurler variant [MPSIH]). However, this …

Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …

To date, almost 2600 gene therapy clinical trials have been completed, are ongoing or have
been approved worldwide. Our database brings together global information on gene therapy …

Alzheimer's disease (AD) and treatment of the brain in aging require the development of
new biologic drugs, such as recombinant proteins or gene therapies. Biologics are large …