The results support the potential of AAV-Parkin gene therapy as a disease-modifying approach for Parkin-deficient EOPD and developed a translatable gene therapy approach using rodents.
This work generates human induced pluripotent stem cell-derived midbrain dopaminergic neurons using both a 2D differentiation protocol and a recently developed 3D method based on the microencapsulation of hiPSCs in small alginate/fibronectin beads.
A. Zanon, E. Kerschbamer, D. Riekschnitz et al.· Communications Biology· 0 citations
Findings support the protective potential of RTA-408 in SCA3-related models and show its effects were associated with activation of Nrf2-related antioxidant responses, p62 upregulation, and selected improvements in mitochondrial phenotypes.
Shin-Hung Pan, Jui-Chih Chang, Wan-Hsuan Lin et al.· Frontiers in Pharmacology· 0 citations
Evidence is presented that the strategy of supporting the ALP system by overexpressing BAG3 has potential therapeutic use for myodegenerative conditions associated with abnormal protein aggregates and mitochondrial turnover.
B. Ozes, Ling-Ying Tong, M. Myers et al.· Human Gene Therapy· 0 citations
It is suggested that BL-918 could potentially modulate SMP30 at the functional protein level, and SwissADME and ProTox 3.0 characterized BL-918 as a viable therapeutic lead, outlining key pharmacokinetic targets for future optimization.
Hardi M. Makwana, S. S. Swain, B. Paital et al.· Journal of Applied Pharmaceu...· 0 citations
In vivo proof-of-concept for SNCA-targeted transcriptional repression therapy in a PD-mouse model is provided and its further preclinical development toward investigational new drug enablement is supported.
Bernadette O'Donovan, Joseph E. Rittiner, Suraj Upadhya et al.· Neurotherapeutics· 0 citations
It is demonstrated that physiologically regulated FXN replacement is sufficient to achieve substantial functional rescue in FA, supporting a gene therapy strategy based on a transgene expression driven by endogenous regulatory elements.
Federica Pilotto, Laure Dall'Agnol, L. Reutenauer et al.· Human Gene Therapy· 0 citations
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