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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency.
Pham V, Tricoli L, Hong X, Wongkittichote P, Castruccio Castracani C, Guerra A, Schlotawa L, Adang LA, Kuhs A, Cassidy MM, Kane O, Tsai E, Presa M, Lutz C, Rivella SB, Ahrens-Nicklas RC. Pham V, et al. Among authors: rivella sb. Mol Ther. 2024 Nov 6;32(11):3829-3846. doi: 10.1016/j.ymthe.2024.08.015. Epub 2024 Aug 22. Mol Ther. 2024. PMID: 39169621
Use of HSC-targeted LNP to generate a mouse model of lethal α-thalassemia and treatment via lentiviral gene therapy.
Chappell ME, Breda L, Tricoli L, Guerra A, Jarocha D, Castruccio Castracani C, Papp TE, Tanaka N, Hamilton N, Triebwasser MP, Ghiaccio V, Fedorky MT, Gollomp KL, Bochenek V, Roche AM, Everett JK, Cook EJ, Bushman FD, Teawtrakul N, Glentis S, Kattamis A, Mui BL, Tam YK, Weissman D, Abdulmalik O, Parhiz H, Rivella S. Chappell ME, et al. Among authors: rivella s. Blood. 2024 Oct 10;144(15):1633-1645. doi: 10.1182/blood.2023023349. Blood. 2024. PMID: 38949981
Effective Gene Therapy for Metachromatic Leukodystrophy Achieved with Minimal Lentiviral Genomic Integrations.
Tricoli L, Sase S, Hacker J, Pham V, Smith S, Chappell M, Breda L, Hurwitz S, Tanaka N, Castracani CC, Guerra A, Hou Z, Schlotawa L, Radhakrishnan K, Kurre P, Ahrens-Nicklas R, Adang L, Vanderver A, Rivella S. Tricoli L, et al. Among authors: rivella s. bioRxiv [Preprint]. 2024 Mar 14:2024.03.14.584404. doi: 10.1101/2024.03.14.584404. bioRxiv. 2024. PMID: 38559013 Free PMC article. Preprint.
Iron restriction in sickle cell disease: When less is more.
Castro OL, De Franceschi L, Ganz T, Kanter J, Kato GJ, Pasricha SR, Rivella S, Wood JC. Castro OL, et al. Among authors: rivella s. Am J Hematol. 2024 Jul;99(7):1349-1359. doi: 10.1002/ajh.27267. Epub 2024 Feb 23. Am J Hematol. 2024. PMID: 38400590 Review.
158 results