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 W. Daniels; Beverly Ann Jenkins; Ellen C Furber; Milankumar Kothiya; Michael B. Banjoko; Raghu Gowda; Jeremiah Hendricks; Yi‐Ya Fang; Priyam Raut; Adnan Arnaout; Fares Bassil; LiChin Wong; Garrett Daniels; Jorge F. Haller; Sid Kamalakaran; Travis “Pete” Lewis; Theresa A. Day; Benjamin Shykind; Mansuo Shannon; Franz Hefti; Anindya Sen

doi:10.1212/wnl.0000000000204897

Abstract

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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Masoudi et al. (2024) studied this question.

synapsesocial.com/papers/68e6fcc5b6db643587677432 https://doi.org/10.1212/wnl.0000000000204897

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

Abstract

Discussion

Cite This Study

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

Abstract

Discussion

Cite This Study

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