Avalotcagene ontaparvovec
Avalotcagene ontaparvovec (DTX301) is "a non-replicating, recombinant self-complimentary adeno-associated virus vector serotype 8 (scAAV8)-encoding human ornithine transcarbamylase". It is developed by Dimension Therapeutics for ornithine transcarbamylase (OTC) deficiency.[1][2][3]
References
[edit]- ^ Wang, Lili; Warzecha, Claude C.; Kistner, Alexander; Chichester, Jessica A.; Bell, Peter; Buza, Elizabeth L.; He, Zhenning; Pampena, M. Betina; Couthouis, Julien; Sethi, Sunjay; McKeever, Kathleen; Betts, Michael R.; Kakkis, Emil; Wilson, James M.; Wadsworth, Samuel; Sullivan, Barbara A. (2022). "Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression". Molecular Therapy - Methods & Clinical Development. 24. Elsevier BV: 292–305. doi:10.1016/j.omtm.2022.01.007. ISSN 2329-0501. PMC 8841522. PMID 35211641.
- ^ Kistner, Alexander; Chichester, Jessica A.; Wang, Lili; Calcedo, Roberto; Greig, Jenny A.; Cardwell, Leah N.; Wright, Margaret C.; Couthouis, Julien; Sethi, Sunjay; McIntosh, Brian E.; McKeever, Kathleen; Wadsworth, Samuel; Wilson, James M.; Kakkis, Emil; Sullivan, Barbara A. (2023-10-13). "Prednisolone and rapamycin reduce the plasma cell gene signature and may improve AAV gene therapy in cynomolgus macaques". Gene Therapy. 31 (3–4). Springer Science and Business Media LLC: 128–143. doi:10.1038/s41434-023-00423-z. ISSN 0969-7128. PMC 10940161. PMID 37833563.
- ^ Nathwani, Amit C.; McIntosh, Jenny; Sheridan, Rose (1 September 2022). "Liver Gene Therapy". Human Gene Therapy. 33 (17–18): 879–888. doi:10.1089/hum.2022.169. PMID 36082993. S2CID 252160990.