Therapeutic delivery of recombinant glucocerebrosidase enzyme-containing extracellular vesicles to human cells from Gaucher disease patients
Summary
This study presents a promising therapeutic strategy to treat severe types of LSDs. It involves delivering lysosomal enzymes to the endocytic compartment of human cells affected by conditions such as GDs with neurological symptoms, as well as potentially other neurological disorders impacting lysosomes. © 2024. The Author(s).
| Authors | Janpipatkul K, Sutjarit N, Tangprasittipap A, Chaiamarit T, Innachai P, Suksen K, Chokpanuwat T, Tim-Aroon T, Anurathapan U, Jearawiriyapaisarn N, Tubsuwan A, Bowornpinyo S, Asavapanumas N, Chairoungdua A, Bhukhai K, Hongeng S |
|---|---|
| Journal | Orphanet journal of rare diseases |
| Publication Date | 2024 Oct 2;19(1):363 |
| PubMed | 39358794 |
| PubMed Central | PMC11445852 |
| DOI | 10.1186/s13023-024-03376-7 |