The Canavan Research Fund of National Tay-Sachs & Allied Diseases Association, Inc. (NTSAD) supports research to identify effective treatments for Canavan disease. Research grants made from this fund will be directed to Canavan research specifically, and when needed will also help defray travel-related clinical trial expenses for affected patients.
What is Canavan Disease?
Canavan disease is a progressive neurological genetic disorder caused by the absence of a vital enzymeAccording to Genome.gov, an enzyme is a biological catalyst and is almost always a protein. It speeds up the rate of a specific chemical reaction in the cell. The enzyme is not destroyed during the reaction and is used over and over. More known as aspartoacylase (ASPA). ASPA breaks N-acetylaspartate acid (NAA) into building blocks essential for building myelinIs the white matter coating our nerves, enabling them to conduct impulses between the brain and other parts of the body. It consists of a layer of proteins packed between two layers of lipids. Myelin is produced by specialized cells: oligodendrocytes in the central nervous system, and Schwann cells in the peripheral nervous system. Myelin sheaths wrap themselves around axons, the threadlike extensions of neurons that make up nerve fibers. Each oligodendrocyte can myelinate several axons. More. Myelin is a fatty membrane (also known as white matter) that forms a proactive coating around each nerve ensuring the nerve functions properly. Canavan disease is one of more than 50 genetically inherited disorders known as leukodystrophies.
Clinical Trials
There are currently two geneOften referred to as the "unit of heredity." A gene is composed of a sequence of DNA required to produce a functional protein. More therapy programs for Canavan disease –
Aspa Therapeutics licensed Dr. Guangping Gao’s Canavan gene therapy work from the University of Massachusetts Chan Medical School in Worcester, Massachusetts.
The other program – Myrtelle, Inc. licensed the research of Paola Leone, PhD, and explores gene therapy using a different approach.