The history of brain drug delivery is reviewed beginning with the first demonstration, in 1914,
that a drug for syphilis, salvarsan, did not enter the brain, due to the presence of a blood …

Abstract Lysosomal Storage Disorders (LSDs) are a diverse group of inherited, monogenic
diseases caused by functional defects in specific lysosomal proteins. The lysosome is a …

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 …

Lysosomal enzyme deficiencies comprise a large group of genetic disorders that generally
lack effective treatments. A potential treatment approach is to engineer the patient's own …

Innate immune factors may restrict hematopoietic stem cell (HSC) genetic engineering and
contribute to broad individual variability in gene therapy outcomes. Here, we show that …

The majority of lysosomal storage diseases affect the brain. Treatment of the brain with
intravenous enzyme replacement therapy is not successful, because the recombinant …

Lysosomal storage disorders (LSDs) are a broad class of monogenic diseases with an
overall incidence of 1: 7,000 newborns, due to the defective activity of one or more …

Lysosomal storage disorders (LSDs) are a group of monogenic diseases characterized by
progressive accumulation of undegraded substrates into the lysosome, due to mutations in …

Abstract Mucopolysaccharidosis I-Hurler (MPS IH) is the most severe form of a metabolic
genetic disease caused by mutations of IDUA gene encoding the lysosomal α-L-iduronidase …

Lysosomal storage diseases (LSDs) are rare inherited metabolic disorders characterized by
a dysfunction in lysosomes, leading to waste material accumulation and severe organ …