Therapeutic delivery of recombinant glucocerebrosidase enzyme-containing extracellular vesicles to human cells from Gaucher disease patients
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.
Authors
- Kanoknetr Suksen (ORCID: https://orcid.org/0000-0003-3130-3920)
- Arthit Chairoungdua (ORCID: https://orcid.org/0000-0002-0965-126X)
- Kanit Bhukhai (ORCID: https://orcid.org/0000-0003-4777-0291)
- Tai Chaiamarit (ORCID: https://orcid.org/0000-0003-0803-1971)
- Nithi Asavapanumas (ORCID: https://orcid.org/0000-0002-5362-2124)
- Natee Jearawiriyapaisarn (ORCID: https://orcid.org/0000-0002-1290-5603)
- Tanida Chokpanuwat
- Suradej Hongeng (ORCID: https://orcid.org/0000-0002-7376-9393)
- Thipwimol Tim‐Aroon (ORCID: https://orcid.org/0000-0001-8044-7993)
- Alisa Tubsuwan (ORCID: https://orcid.org/0000-0003-2412-6102)
- Amornrat Tangprasittipap (ORCID: https://orcid.org/0000-0003-0313-2191)
- Keatdamrong Janpipatkul
- Nareerat Sutjarit (ORCID: https://orcid.org/0000-0002-8152-3684)
- Usanarat Anurathapan (ORCID: https://orcid.org/0000-0002-2791-7471)
- Pawarit Innachai
- Supareak Bowornpinyo
Institutions
- Mahidol University (TH)
- Navamindradhiraj University (TH)
- Vajira Hospital (TH)
- Ramathibodi Hospital (TH)
Publication Details
- Journal
- Orphanet Journal of Rare Diseases
- Published
- 2024-10-02
- DOI
- https://doi.org/10.1186/s13023-024-03376-7
- Citations
- 2
- Primary Topic
- Extracellular vesicles in disease
- Type
- article
- Field-Weighted Citation Impact
- 0.47
Funders
- Mahidol University
- National Research Council of Thailand
- Ramathibodi Foundation
- Faculty of Science, Mahidol University