Deliver CRISPR to the Brain - A New Treatment Strategy for Mucopolysaccharidoses

Mucopolysaccharidosis I (MPS I) is a rare genetic disorder that affects many body systems and leads to organ damage. The disease arises through mutations in the ɑ-L-iduronidase (IDUA) gene, which encodes for an enzyme called alpha-L-iduronidase. Mutations in this gene result in cells that either produce the enzyme in low amounts or cannot produce it at all. The enzyme is needed to break down substances called “glycosaminoglycans” (GAGs), which are by-products of chemical reactions in the body’s cells. If GAGs are not broken down, they accumulate in cells, eventually leading to cell, tissue, and organ damage.

Earlier this year, a Brazilian research team led by Dr. Guilherme BaldoatUniversidade Federal do Rio Grande do Sul, Porto Alegre in Brazil reported the development of a new CRISPR-based method to treat MPS. This method involves nasal delivery of CRISPR gene-editing reagents to the brain, which led to partial restoration of IDUA activity in the brain. So far, the team has demonstrated that this approach can improve cognitive symptoms in a mouse model of MPS. Experiments will proceed with monkeys later this year.

What will you learn from this webinar:

• Characteristics of MPS
• Non-viral delivery of gene-editing reagents to the brain through the nose
• Effects of gene editing on MPS

Tentative Webinar Programme:

• 15.00 Welcome and introduction by CRISPR Medicine News
• 15.05 Guilherme Baldo, Associate Professor, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil | Deliver CRISPR to the Brain - A New Treatment Strategy for Mucopolysaccharidoses
• 15.50 Q & A with Guilherme Baldo
• 16.25 Close by CRISPR Medicine News

Speaker | Title:

Guilherme Baldo, Associate Professor, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil | Deliver CRISPR to the Brain - A New Treatment Strategy for Mucopolysaccharidoses

CMN interview

Guilherme Baldo, Associate Professor, Universidade Federal do Rio Grande do Sul, Porto Alegre, Brazil

Dr. Baldo is an associate professor at the Federal University of Rio Grande do Sul in Porto Alegrel (UFRGS), Brazil. Here, he is working on the development of innovative treatments for rare genetic diseases, with a particular focus on gene therapy and genome editing. He has authored more than 80 articles published in international journals, with a research productivity scholarship from CNPq level 2. Moreover, he is Professor of the Graduate Programs in Physiology and in Genetics and Molecular Biology at UFRGS, having supervised 15 ongoing or completed masters and doctoral theses. Dr. Baldo speaks at courses and events in the country and abroad on the topic of advanced therapies.

Read our CMN interview with Dr. Baldo from March 2022.