Microglial-targeted Gene Therapy: Developing a Disease Modifying Treatment for ALSP Associated with CSF1R Mutations (ALSP-CSF1R) (P11-4.012)

Neda Masoudi, Jessie Willen, Carter Daniels, Beverly Ann Jenkins, Ellen Conceicao Furber, Milankumar Kothiya, Michael B. Banjoko, Raghu Gowda,Jeremiah Hendricks, Yi-Ya Fang,Priyam Raut, Adnan Arnaout, Fares Bassil, LiChin Wong, Garrett Daniels,Jorge Francisco Haller,Sid Kamalakaran, Travis Lewis,Theresa Ann Day, Benjamin Shykind, Mansuo Shannon,Franz Hefti, Anindya Sen

Neurology（2024）

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摘要

We are developing PR009 (LY3884965), a rAAV gene therapy, as a disease-modifying, one-time treatment for ALSP-CSF1R. PR009 is designed to enhance microglial function in patients' brains by increasing TREM2 (Triggering receptor expressed on myeloid cells-2) levels.

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