The formulation and delivery of biopharmaceutical drugs, such as monoclonal antibodies
and recombinant proteins, poses substantial challenges owing to their large size and …

Introduction: There are 2 misconceptions about the cerebrospinal fluid (CSF), the blood-
brain barrier (BBB), and brain drug delivery, which date back to the discovery of a barrier …

To what extent does the subarachnoid cerebrospinal fluid (CSF) compartment communicate
directly with the extravascular compartment of human brain tissue? Interconnection between …

The neurons of the central nervous system (CNS) require precise control of their bathing
microenvironment for optimal function, and an important element in this control is the blood …

Mucopolysaccharidosis type II (MPS II, Hunter syndrome) was first described by Dr. Charles
Hunter in 1917. Since then, about one hundred years have passed and Hunter syndrome …

Mucopolysaccharidosis II (MPS II) is an X-linked recessive lysosomal storage disease
caused by mutations in the iduronate-2-sulfatase (IDS) gene. Since IDS catalyzes the …

In 1964, Christian de Duve first suggested that enzyme replacement might prove therapeutic
for lysosomal storage diseases (LSDs). Early efforts identified the major obstacles, including …

Lysosomes and lysosomal enzymes play a central role in numerous cellular processes,
including cellular nutrition, recycling, signaling, defense, and cell death. Genetic deficiencies …

Intrathecal delivery methods have been used for many decades to treat a broad range of
central nervous system disorders. A literature review demonstrated that …

Initially thought to be useful only to reach tissues in the immediate vicinity of the CSF
circulatory system, CSF circulation is now increasingly viewed as a viable pathway to deliver …