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MDS 2022 | The impact of wild-type HTT suppression on brain function

Rajeev Kumar, MD, Rocky Mountain Movement Disorders Center, Englewood, CO, and Ann Morgan, PhD, Annexon Biosciences, discuss the impact of wild-type huntingtin (HTT) suppression with mutant HTT lowering approaches. There is growing concern that wild-type HTT may have an important role in normal brain functions, even though high degrees of wild-type HTT knockdown seem to be well-tolerated in animal models of Huntington’s disease. It is hypothesized that the significant knockdown of wild-type HTT may play a role in some of the adverse effects observed in the tominersen trial. Therefore, the preservation of wild-type HTT may be important for normal brain function in patients with Huntington’s disease, but further research is needed. This interview took place at the 2022 International Congress of Parkinson’s Disease and Movement Disorders in Madrid, Spain.

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Disclosures

Dr Morgan is an employee of Annexon Biosciences.
Dr Kumar is a consultant for Annexon Biosciences, and his institution receives funding from Annexon Biosciences.