In vivo target-engagement studies have demonstrated that delivering GBA1 transgenes using a BBB-penetrant AAV capsid resulted in therapeutically relevant levels of GCase in multiple brain regions ...

Covariance matrices can be used to determine dynamic information such as DFI and DCI. Matrices are located in .rar files corresponding to each tested variant, with "cleaned" indicating the wild type.

“The potential of GCase as a therapeutic target beyond Parkinson’s Disease is an encouraging finding that we can incorporate into our future development plans for GT-02287.” The late ...

Capsida Biotherapeutics Inc. presented preclinical data for a new next-generation gene supplementation therapy candidate, CAP-003, for Parkinson’s disease (PD) patients with GBA1 mutations (PD-GBA).

3 Asan Institute for Life Sciences, Asan Medical Center, College of Medicine University of Ulsan, Seoul, The Republic of Korea Background Ambroxol (ABX) has been suggested as an augmentative ...

Mutations in GBA, the gene expressing the GCase enzyme, affect up to 15% of PD patients and are the most common genetic risk factor for PD. Post-mortem studies demonstrate an approximate 30% GCase ...

CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs). These data were presented in a late-breaking poster ...

CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs). These data were presented in a late-breaking poster ...

J.P. Morgan analyst Tessa Romero downgraded the rating on Neumora Therapeutics, Inc. (NMRA – Research Report) to a Hold today, setting a ...