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Year Number of Results
2006 4
2007 2
2008 1
2009 1
2012 1
2014 1
2015 1
2016 1
2017 3
2018 1
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2022 3
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51 results

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Page 1
Lentiviral Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan CN, Musolino PL, Lund TC, Gupta AO, De Oliveira S, Thrasher AJ, Aubourg P, Kühl JS, Loes DJ, Amartino H, Smith N, Folloni Fernandes J, Sevin C, Sankar R, Hussain SA, Gissen P, Dalle JH, Platzbecker U, Downey GF, McNeil E, Demopoulos L, Dietz AC, Thakar HL, Orchard PJ, Williams DA. Eichler F, et al. Among authors: musolino pl. N Engl J Med. 2024 Oct 10;391(14):1302-1312. doi: 10.1056/NEJMoa2400442. N Engl J Med. 2024. PMID: 39383459 Free PMC article. Clinical Trial.
COL4A1 and COL4A2-related disorders: Clinical features, diagnostic guidelines, and management.
Tambala D, Vassar R, Snow J, Balestrini S, Bersano A, Guey S, Bonaventura E, Signorini S, Sartori S, Bertini E, Tonduti D, Parazzini C, Macchiaiolo M, Pelizza MF, Pichiecchio A, Massella L, Coste T, Orcesi S, Politano D, Bacci G, Marziali E, Dollfus H, Mandelli A, Chinali M, Plaisier E, Simioni P, Colombatti R, Guerrini R, Tournier-Lasserve E, Gould DB, Musolino PL. Tambala D, et al. Among authors: musolino pl. Genet Med. 2025 Sep;27(9):101514. doi: 10.1016/j.gim.2025.101514. Epub 2025 Jul 2. Genet Med. 2025. PMID: 40616396 Free article.
Immune evasive DNA donors and recombinases license kilobase-scale writing.
Tou CJ, Xie K, Ferreira da Silva J, Kalailingam P, Amar-Lewis E, Rufino-Ramos D, Sawyer W, Eller ML, Starzyk J, Majumdar I, Wang J, Lee D, Yang S, Meis RJ, Dahl GA, Li J, Shan R, Artzi N, Musolino PL, Wu H, Kleinstiver BP. Tou CJ, et al. Among authors: musolino pl. Nature. 2026 May;653(8114):576-586. doi: 10.1038/s41586-026-10241-z. Epub 2026 Mar 11. Nature. 2026. PMID: 41813887
An Engineered Adeno-Associated Virus Capsid Mediates Efficient Transduction of Pericytes and Smooth Muscle Cells of the Brain Vasculature.
Ramirez SH, Hale JF, McCarthy S, Cardenas CL, Dona KNUG, Hanlon KS, Hudry E, Cruz D, Ng C, Das S, Nguyen DM, Nammour J, Bennett RE, Andrews AM, Musolino PL, Maguire CA. Ramirez SH, et al. Among authors: musolino pl. Hum Gene Ther. 2023 Aug;34(15-16):682-696. doi: 10.1089/hum.2022.211. Hum Gene Ther. 2023. PMID: 37376759 Free PMC article.
In vivo Treatment of a Severe Vascular Disease via a Bespoke CRISPR-Cas9 Base Editor.
Alves CRR, Das S, Krishnan V, Ha LL, Fox LR, Stutzman HE, Shamber CE, Kalailingam P, McCarthy S, Lino Cardenas CL, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Lawton J, Kim N, Silverstein RA, da Silva JF, de la Cruz D, Richa R, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME, Kleinstiver BP, Musolino PL. Alves CRR, et al. Among authors: musolino pl. bioRxiv [Preprint]. 2024 Nov 11:2024.11.11.621817. doi: 10.1101/2024.11.11.621817. bioRxiv. 2024. Update in: Nat Biomed Eng. 2026 May;10(5):952-967. doi: 10.1038/s41551-025-01499-1. PMID: 39605323 Free PMC article. Updated. Preprint.
Emergence of the natural history of Myhre syndrome: 47 patients evaluated in the Massachusetts General Hospital Myhre Syndrome Clinic (2016-2023).
Lin AE, Scimone ER, Thom RP, Balaguru D, Kinane TB, Moschovis PP, Cohen MS, Tan W, Hague CD, Dannheim K, Levitsky LL, Lilly E, DiGiacomo DV, Masse KM, Kadzielski SM, Zar-Kessler CA, Ginns LC, Neumeyer AM, Colvin MK, Elder JS, Learn CP, Mou H, Weagle KM, Buch KA, Butler WE, Alhadid K, Musolino PL, Sultana S, Bandyopadhyay D, Rapalino O, Peacock ZS, Chou EL, Heidary G, Dorfman AT, Morris SA, Bergin JD, Rayment JH, Schimmenti LA, Lindsay ME; MGH Myhre Syndrome Study Group. Lin AE, et al. Among authors: musolino pl. Am J Med Genet A. 2024 Oct;194(10):e63638. doi: 10.1002/ajmg.a.63638. Epub 2024 May 23. Am J Med Genet A. 2024. PMID: 38779990 Free PMC article.
Treatment of a severe vascular disease using a bespoke CRISPR-Cas9 base editor in mice.
Alves CRR, Das S, Krishnan V, Ha LL, Fox LR, Stutzman HE, Shamber CE, Kalailingam P, McCarthy S, Lino Cardenas CL, Fong CE, Imai T, Mitra S, Yun S, Wood RK, Benning FMC, Roh K, Lawton J, Kim N, Silverstein RA, Ferreira da Silva J, de la Cruz D, Richa R, Xie J, Gray-Edwards HL, Malhotra R, Chung DY, Chao LH, Tsai SQ, Maguire CA, Lindsay ME, Kleinstiver BP, Musolino PL. Alves CRR, et al. Among authors: musolino pl. Nat Biomed Eng. 2026 May;10(5):952-967. doi: 10.1038/s41551-025-01499-1. Epub 2025 Sep 11. Nat Biomed Eng. 2026. PMID: 40935887 Free PMC article.
Towards precision medicine for brain arteriovenous malformations.
Hale AT, Kundishora AJ, Kalailingam P, Barak T, Duy PQ, Ramundo CM, Fan B, Li Q, Brastianos PK, Shankar GM, Alper SL, Kleinstiver BP, Musolino PL, Kahle KT. Hale AT, et al. Among authors: musolino pl. J Clin Invest. 2026 May 15;136(10):e205086. doi: 10.1172/JCI205086. eCollection 2026 May 15. J Clin Invest. 2026. PMID: 42138076 Free PMC article. Review.
Use of Brain MRI in Cerebral Adrenoleukodystrophy: International Recommendations for Screening, Monitoring, and Research.
Yska HAF, Golse M, Galanaud D, Amartino HM, Bergner C, Bruschi F, Eichler FS, Fatemi A, García-Cazorla À, Gomez-Chiari M, Köhler W, Loes D, Lund T, Mallack EJ, Moscatelli M, Musolino PL, Nascene DR, Orthmann-Murphy JL, Parazzini C, Pouwels PJW, Ribeiro-Constante J, Roosendaal SD, Salsano E, Sgobbi PV, Sevin C, Smith Fine A, Tonduti D, Van Haren K, Zerem A, Engelen M, Mochel F. Yska HAF, et al. Among authors: musolino pl. Neurology. 2026 Mar 10;106(5):e214657. doi: 10.1212/WNL.0000000000214657. Epub 2026 Feb 6. Neurology. 2026. PMID: 41650360 Free PMC article.
51 results