A patient-derived amyotrophic lateral sclerosis blood-brain barrier model for focused ultrasound-mediated anti-TDP-43 antibody delivery.

ABSTRACT:

Background

Amyotrophic lateral sclerosis (ALS) is a rapidly progressing neurodegenerative disorder with minimally effective treatment options. An important hurdle in ALS drug development is the non-invasive therapeutic access to the motor cortex currently limited by the presence of the blood-brain barrier (BBB). Focused ultrasound and microbubble (FUS^+ MB) treatment is an emerging technology that was successfully used in ALS patients to temporarily open the cortical BBB. However, FUS^+ MB-mediated drug delivery across ALS patients' BBB has not yet been reported. Similarly, the effects of FUS^+ MB on human ALS BBB cells remain unexplored.

Methods

Here we established the first FUS^+ MB-compatible, fully-human ALS patient-cell-derived BB

SUBMITTER: Wasielewska JM 

PROVIDER: S-EPMC11323367 | biostudies-literature | 2024 Aug

REPOSITORIES: biostudies-literature