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News From The Journal Of Clinical Investigation: June 3, 2013

A potential for Mucopolysaccharidosis Type IIIA

Mucopolysaccharidosis Type IIIA (MPSIIIA) is a metabolic disorder in which the body is missing an enzyme that is required to break down long chains of sugars known as glycosaminoglycans. Over time, the glycosaminoglycans collect in the body and cause damage, particularly in the brain. In this issue of the , Fàtima Bosch and colleagues at Universitat Autònoma de Barcelona in Spain developed a form of gene therapy to replace the enzyme that is missing in MPSIIIA. By injecting the replacement gene into the the that surrounds the brain and spinal cord, Bosch and colleagues found that they could successfully deliver a replacement gene to the brain in mice and dogs. This study demonstrates that gene therapy can be delivered to the brain through the and suggests that this approach could potentially be used as a therapy for MPSIIIA.

TITLE: Whole body correction of Mucopolysaccharidosis IIIA by intra-cerebrospinal fluid gene therapy


A new target in castration-resistant

The prostate gland requires male hormones, known as androgens, in order to function. Androgens act through cell surface receptors (androgen receptors) that initiate changes in prostate cells. In prostate cancer, androgens promote the growth and spread of cancer cells; consequently, therapeutics that block androgen receptors are effective in many prostate cancer patients. Unfortunately, the disease frequently recurs in a lethal, androgen-independent form (CRPC) that is associated with mutations in androgen receptors. In this issue of the Journal of Clinical Investigation, Marianne Sadar and colleagues at the BC Cancer Agency in Vancouver, British Columbia, investigated a drug, EPI-001, which blocks the activity of the mutant androgen receptors through interaction with a region of the receptor known as the N-terminal domain. Using a mouse model of prostate cancer, Sadar and colleagues found that EPI-001 reduced the growth of prostate cancer. These findings suggest that drugs similar to EPI-001 could potentially be used to treat androgen-independent forms of prostate cancer.

TITLE: An androgen receptor N-terminal domain antagonist for treating prostate cancer



Journal of Clinical Investigation