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Page 1
TRPML-1 Dysfunction and Renal Tubulopathy in Mucolipidosis Type IV.
Grieco G, Montefusco S, Nusco E, Capuozzo A, Cervellini F, Polishchuk E, Bishop M, Miele A, D'Apolito L, La Vecchia C, Aurilia M, Schiavo M, Staiano L, Cesana M, Oberman R, Lynch AV, Musolino P, Trepiccione F, Grishchuk Y, Medina DL. Grieco G, et al. Among authors: musolino p. J Am Soc Nephrol. 2024 Dec 4. doi: 10.1681/ASN.0000000567. Online ahead of print. J Am Soc Nephrol. 2024. PMID: 39630508
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. PMID: 39605323 Free PMC article. Preprint.
Exploring the Relationship Between Radiation-Induced Moya Moya Syndrome and Radiation Dose for Pediatric Patients Treated with Proton Radiation Therapy: Moya Moya in Peds Patients After Proton RT.
Gill GS, Baliga S, Lewy J, Bajaj BV, Gallotto SL, Fitzpatrick A, Patteson B, Speroni S, Omsberg A, Caruso P, Rapalino O, Musolino P, Yock TI. Gill GS, et al. Among authors: musolino p. Int J Radiat Oncol Biol Phys. 2024 Nov 14:S0360-3016(24)03639-3. doi: 10.1016/j.ijrobp.2024.11.070. Online ahead of print. Int J Radiat Oncol Biol Phys. 2024. PMID: 39549759
A Novel Mouse Model for Cerebral Inflammatory Demyelination in X-Linked Adrenoleukodystrophy: Insights into Pathogenesis and Potential Therapeutic Targets.
Hashemi E, Srivastava IN, Aguirre A, Yoseph ET, Kaushal E, Awani A, Ryu JK, Akassoglou K, Talebian S, Chu P, Pisani L, Musolino P, Steinman L, Doyle K, Robinson WH, Sharpe O, Cayrol R, Orchard PJ, Lund T, Vogel H, Lenail M, Han MH, Bonkowsky JL, Van Haren KP. Hashemi E, et al. Among authors: musolino p. Ann Neurol. 2024 Oct 28. doi: 10.1002/ana.27117. Online ahead of print. Ann Neurol. 2024. PMID: 39467011
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.
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 Clinical Trial.
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 calcific arterial disease via enhancement of autophagy using GSK343.
Lino Cardenas CL, Jiang W, Kajuluri LP, Singh K, Ostrom K, Li R, Cherbonneau F, Boerboom S, Birchenough C, Roh K, Chou EL, Shahrooz Z, Nicholson C, Johnson AL, Lee S, Ichinose F, Bloch DB, Nigwekar S, Ellinor PT, Musolino P, Lindsay ME, Dou Z, Miller CL, Malhotra R. Lino Cardenas CL, et al. Among authors: musolino p. iScience. 2023 Oct 29;26(11):108360. doi: 10.1016/j.isci.2023.108360. eCollection 2023 Nov 17. iScience. 2023. PMID: 38033629 Free PMC article.
A novel mouse model of cerebral adrenoleukodystrophy highlights NLRP3 activity in lesion pathogenesis.
Hashemi E, Narain Srivastava I, Aguirre A, Tilahan Yoseph E, Kaushal E, Awani A, Kyu Ryu J, Akassoglou K, Talebian S, Chu P, Pisani L, Musolino P, Steinman L, Doyle K, Robinson WH, Sharpe O, Cayrol R, Orchard P, Lund T, Vogel H, Lenail M, Han MH, Bonkowsky JL, Van Haren KP. Hashemi E, et al. Among authors: musolino p. bioRxiv [Preprint]. 2023 Nov 10:2023.11.07.564025. doi: 10.1101/2023.11.07.564025. bioRxiv. 2023. PMID: 37986739 Free PMC article. Preprint.
52 results