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Phase 1 Study of AAV9.LAMP2B Gene Therapy in Danon Disease.
Greenberg B, Taylor M, Adler E, Colan S, Ricks D, Yarabe P, Battiprolu P, Shah G, Patel K, Coggins M, Carou-Keenan S, Schwartz JD, Rossano JW. Greenberg B, et al. Among authors: ricks d. N Engl J Med. 2024 Nov 18. doi: 10.1056/NEJMoa2412392. Online ahead of print. N Engl J Med. 2024. PMID: 39556016
AAV-Mediated Delivery of Plakophilin-2a Arrests Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in Murine Hearts: Preclinical Evidence Supporting Gene Therapy in Humans.
van Opbergen CJM, Narayanan B, Sacramento CB, Stiles KM, Mishra V, Frenk E, Ricks D, Chen G, Zhang M, Yarabe P, Schwartz J, Delmar M, Herzog CD, Cerrone M. van Opbergen CJM, et al. Among authors: ricks d. Circ Genom Precis Med. 2024 Feb;17(1):e004305. doi: 10.1161/CIRCGEN.123.004305. Epub 2024 Jan 30. Circ Genom Precis Med. 2024. PMID: 38288614 Free article.
Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal.
Moscatelli I, Almarza E, Schambach A, Ricks D, Schulz A, Herzog CD, Henriksen K, Askmyr M, Schwartz JD, Richter J. Moscatelli I, et al. Among authors: ricks d. Mol Ther Methods Clin Dev. 2020 Dec 25;20:389-397. doi: 10.1016/j.omtm.2020.12.009. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33575431 Free PMC article. Review.
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