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Year Number of Results
2006 4
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62 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.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, Armant M, Dansereau C, Lund TC, Miller WP, Raymond GV, Sankar R, Shah AJ, Sevin C, Gaspar HB, Gissen P, Amartino H, Bratkovic D, Smith NJC, Paker AM, Shamir E, O'Meara T, Davidson D, Aubourg P, Williams DA. Eichler F, et al. Among authors: musolino pl. N Engl J Med. 2017 Oct 26;377(17):1630-1638. doi: 10.1056/NEJMoa1700554. Epub 2017 Oct 4. N Engl J Med. 2017. PMID: 28976817 Free PMC article. Clinical Trial.
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.
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.
Intensive vs Standard Treatment of Hyperglycemia and Functional Outcome in Patients With Acute Ischemic Stroke: The SHINE Randomized Clinical Trial.
Johnston KC, Bruno A, Pauls Q, Hall CE, Barrett KM, Barsan W, Fansler A, Van de Bruinhorst K, Janis S, Durkalski-Mauldin VL; Neurological Emergencies Treatment Trials Network and the SHINE Trial Investigators. Johnston KC, et al. JAMA. 2019 Jul 23;322(4):326-335. doi: 10.1001/jama.2019.9346. JAMA. 2019. PMID: 31334795 Free PMC article. Clinical Trial.
Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy.
Lauer A, Speroni SL, Choi M, Da X, Duncan C, McCarthy S, Krishnan V, Lusk CA, Rohde D, Hansen MB, Kalpathy-Cramer J, Loes DJ, Caruso PA, Williams DA, Mouridsen K, Emblem KE, Eichler FS, Musolino PL. Lauer A, et al. Among authors: musolino pl. Nat Commun. 2023 Apr 5;14(1):1900. doi: 10.1038/s41467-023-37262-w. Nat Commun. 2023. PMID: 37019892 Free PMC article.
Endovascular thrombectomy for childhood stroke (Save ChildS Pro): an international, multicentre, prospective registry study.
Sporns PB, Bhatia K, Abruzzo T, Pabst L, Fraser S, Chung MG, Lo W, Othman A, Steinmetz S, Jensen-Kondering U, Schob S, Kaiser DPO, Marik W, Wendl C, Kleffner I, Henkes H, Kraehling H, Nguyen-Kim TDL, Chapot R, Yilmaz U, Wang F, Hafeez MU, Requejo F, Limbucci N, Kauffmann B, Möhlenbruch M, Nikoubashman O, Schellinger PD, Musolino P, Alawieh A, Wilson J, Grieb D, Gersing AS, Liebig T, Olivieri M, Schwabova JP, Tomek A, Papanagiotou P, Boulouis G, Naggara O, Fox CK, Orlov K, Kuznetsova A, Parra-Farinas C, Muthusami P, Regenhardt RW, Dmytriw AA, Burkard T, Martinez M, Brechbühl D, Steinlin M, Sun LR, Hassan AE, Kemmling A, Lee S, Fullerton HJ, Fiehler J, Psychogios MN, Wildgruber M. Sporns PB, et al. Among authors: musolino p. Lancet Child Adolesc Health. 2024 Dec;8(12):882-890. doi: 10.1016/S2352-4642(24)00233-5. Epub 2024 Oct 11. Lancet Child Adolesc Health. 2024. PMID: 39401507 Free article. Clinical Trial.
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. 2025 Sep 11. doi: 10.1038/s41551-025-01499-1. PMID: 39605323 Free PMC article. Updated. Preprint.
The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy.
Rodríguez-Pascau L, Vilalta A, Cerrada M, Traver E, Forss-Petter S, Weinhofer I, Bauer J, Kemp S, Pina G, Pascual S, Meya U, Musolino PL, Berger J, Martinell M, Pizcueta P. Rodríguez-Pascau L, et al. Among authors: musolino pl. Sci Transl Med. 2021 Jun 2;13(596):eabc0555. doi: 10.1126/scitranslmed.abc0555. Sci Transl Med. 2021. PMID: 34078742
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. 2025 Sep 11:10.1038/s41551-025-01499-1. doi: 10.1038/s41551-025-01499-1. Online ahead of print. Nat Biomed Eng. 2025. PMID: 40935887 Free PMC article.
62 results