Gene Therapy Iniative
“Gene therapy is an exciting avenue that may pave the way to new and innovative treatment possibilities and may help reduce the impact of the third copy of chromosome 21 in people with Down syndrome.”
Our Gene Therapy in Down Syndrome effort is a longer term research initiative for LuMind IDSC that builds on novel therapeutic target discoveries and the major momentum in the gene therapy field. The field has seen dramatic progress in the last 10 years, including several FDA approved therapies and novel delivery techniques and manufacturing capabilities. Multiple genes have been identified on chromosome 21 that are targets for therapeutic intervention, including for Alzheimer (APP, BACE2), Inflammation (Interferon-receptor genes) and Cognition (DYRK1a, oxidative-stress gees). Also, the gene XIST naturally silences one of the two copies of chromosome X and research is ongoing to target the silencing of the 3rd copy of chromosome 21 with the XIST gene.
Why it Matters
Gene therapy is an exciting avenue that may pave the way to new and innovative treatment possibilities and may help reduce the impact of the third copy of chromosome 21 in people with Down syndrome. For example, it would be great to be able to silence the extra Alzheimer’s genes and delay the onset of Alzheimer’s disease by 20 years or more. And a gene therapy that slightly helps improve memory and cognition could mean greater learning at school, improved daily living independence and broader career choices and opportunities.
What LuMind IDSC is Doing
LuMind IDSC is working on the following:
- Funding and following research in understanding the role of specific genes for disease in Down syndrome is an important goal to LuMind IDSC;
- Held expert meeting on gene therapy in Down syndrome to build a roamap towards cinical trials.
- Supporting gene therapy approaches, that have the potential to identify genes of interest as well as to conduct gene therapy in the Down syndrome population.
- Attending gene therapy conferences for a deeper understanding of the field.
How You Can Help
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