The Walia Lab focuses our efforts on developing gene therapies to treat disorders of the central nervous system.
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Tay Sach's and Sandhoff's Disease
Tay Sach's and Sandhoff's disease are part of a family of disorders known as GM2 Gangliosidosis, a type of rare inherited lysosomal storage disorder. They can occur when mutations arise in the HEXA or HEXB genes respectively. This results in the deficiency of the HexA enzyme leading to a severe neurological phenotype. The Walia lab focuses on optimizing gene therapy approaches for treating these diseases.
AB Variant and The Role of GM2AP
Another form of GM2 gangliosidosis is known as AB Variant (ABGM2). ABGM2 is characterized by a mutation in the GM2A gene that encodes the GM2-activator protein (GM2AP), which is an essential co-factor in the breakdown of GM2 gangliosides. The inability to synthesize properly functioning GM2AP leads to a buildup of GM2 gangliosides ultimately leading to cell death. The Walia Lab is looking to investigate a gene therapy approach to treating ABGM2 and producing a more accurate mouse model of the disease. We are also interested in understanding the role that GM2AP may play in other diseases such as cancer and atherosclerosis.