Marisa Wexler, MS, senior science writer —

Marisa holds a Master of Science in cellular and molecular pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. Her areas of expertise include cancer biology, immunology, and genetics, and she has worked as a science writing and communications intern for the Genetics Society of America.

Articles by Marisa Wexler

Sanfilippo type A children show gains with gene therapy UX111

Most children with Sanfilippo syndrome type A who received the one-time gene therapy UX111 early in life in a clinical trial continue to show cognitive development gains beyond the age where patients usually start regressing. These improvements were associated with reductions in heparan sulfate (HS) in the children’s cerebrospinal fluid…

Vision problems may be earliest symptom of Sanfilippo C: Study

Light-sensing cells in the eyes become damaged and die in Sanfilippo syndrome type C before the onset of behavioral symptoms, a new study indicates. Issues related to vision “may be the first truly distinctive symptom” of Sanfilippo for these patients, which suggests “better disease awareness among ophthalmologists could play…

Iminosugars may help advance treatments for Sanfilippo: Cell study

Treatment with sugary molecules called iminosugars can reduce the toxic buildup of heparan sulfate in cellular models of Sanfilippo syndrome, a new study reports. The study, “N-Substituted l-Iminosugars for the Treatment of Sanfilippo Type B Syndrome,” was published in the Journal of Medicinal Chemistry. Sanfilippo syndrome is…

Resveratrol Prevents Toxic Buildup in Cells of Sanfilippo Mouse Model

Treatment with resveratrol, a plant-derived compound that activates a cell cleansing process called autophagy, reduced the toxic buildup of heparan sulfate and normalized behavior in a mouse model of Sanfilippo syndrome. “Resveratrol normalized urinary [heparan sulfate] levels and attenuated abnormal behaviors in [Sanfilippo B] model mice, supporting further studies…

Enzyme Replacement Therapy for Sanfilippo A Shows Promise

A novel enzyme replacement therapy developed by BioMarin Pharmaceutical for Sanfilippo syndrome type A showed promising effects in a mouse model of the disease, a study showed. Researchers were also able to identify a previously unknown receptor protein that lets the enzyme enter cells. The study, “…