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Items: 1 to 50 of 56

1.

Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease.

Stiles KM, Sondhi D, Kaminsky SM, De BP, Rosenberg JB, Crystal RG.

Chronic Obstr Pulm Dis. 2018 Aug 17;5(4):244-257. doi: 10.15326/jcopdf.5.4.2017.0160.

2.

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease).

Sindelar M, Dyke JP, Deeb RS, Sondhi D, Kaminsky SM, Kosofsky BE, Ballon DJ, Crystal RG, Gross SS.

Sci Rep. 2018 Oct 15;8(1):15229. doi: 10.1038/s41598-018-33449-0.

3.

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study.

Nickel M, Simonati A, Jacoby D, Lezius S, Kilian D, Van de Graaf B, Pagovich OE, Kosofsky B, Yohay K, Downs M, Slasor P, Ajayi T, Crystal RG, Kohlschütter A, Sondhi D, Schulz A.

Lancet Child Adolesc Health. 2018 Aug;2(8):582-590. doi: 10.1016/S2352-4642(18)30179-2. Epub 2018 Jul 2. Erratum in: Lancet Child Adolesc Health. 2018 Sep;2(9):e24.

PMID:
30119717
4.

Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema.

Qiu T, Chiuchiolo MJ, Whaley AS, Russo AR, Sondhi D, Kaminsky SM, Crystal RG, Pagovich OE.

Allergy. 2019 Jun;74(6):1081-1089. doi: 10.1111/all.13582. Epub 2019 Mar 19.

PMID:
30059156
5.

In Vivo Potency Assay for Adeno-Associated Virus-Based Gene Therapy Vectors Using AAVrh.10 as an Example.

De BP, Chen A, Salami CO, Van de Graaf B, Rosenberg JB, Pagovich OE, Sondhi D, Crystal RG, Kaminsky SM.

Hum Gene Ther Methods. 2018 Jun;29(3):146-155. doi: 10.1089/hgtb.2017.246. Epub 2018 Jun 8.

PMID:
29706115
6.

Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2.

Markmann S, J Christie-Reid J, Rosenberg JB, De BP, Kaminsky SM, Crystal RG, Sondhi D.

Exp Neurol. 2018 Aug;306:22-33. doi: 10.1016/j.expneurol.2018.04.001. Epub 2018 Apr 12.

PMID:
29655638
7.

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease.

Rosenberg JB, Kaplitt MG, De BP, Chen A, Flagiello T, Salami C, Pey E, Zhao L, Ricart Arbona RJ, Monette S, Dyke JP, Ballon DJ, Kaminsky SM, Sondhi D, Petsko GA, Paul SM, Crystal RG.

Hum Gene Ther Clin Dev. 2018 Mar;29(1):24-47. doi: 10.1089/humc.2017.231. Epub 2018 Mar 13.

8.

Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders.

Markmann S, De BP, Reid J, Jose CL, Rosenberg JB, Leopold PL, Kaminsky SM, Sondhi D, Pagovich O, Crystal RG.

Hum Gene Ther. 2018 Apr;29(4):403-412. doi: 10.1089/hum.2017.203. Epub 2018 Mar 20.

PMID:
29316814
9.

Radioiodinated Capsids Facilitate In Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors.

Kothari P, De BP, He B, Chen A, Chiuchiolo MJ, Kim D, Nikolopoulou A, Amor-Coarasa A, Dyke JP, Voss HU, Kaminsky SM, Foley CP, Vallabhajosula S, Hu B, DiMagno SG, Sondhi D, Crystal RG, Babich JW, Ballon D.

Sci Rep. 2017 Jan 6;7:39594. doi: 10.1038/srep39594.

10.

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice.

Liu W, Zhao L, Blackman B, Parmar M, Wong MY, Woo T, Yu F, Chiuchiolo MJ, Sondhi D, Kaminsky SM, Crystal RG, Paul SM.

J Neurosci. 2016 Dec 7;36(49):12425-12435. Erratum in: J Neurosci. 2017 Mar 29;37(13):3734.

11.

Genetic Modification of the Lung Directed Toward Treatment of Human Disease.

Sondhi D, Stiles KM, De BP, Crystal RG.

Hum Gene Ther. 2017 Jan;28(1):3-84. doi: 10.1089/hum.2016.152. Review.

PMID:
27927014
12.

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma.

Hicks MJ, Chiuchiolo MJ, Ballon D, Dyke JP, Aronowitz E, Funato K, Tabar V, Havlicek D, Fan F, Sondhi D, Kaminsky SM, Crystal RG.

PLoS One. 2016 Oct 6;11(10):e0162978. doi: 10.1371/journal.pone.0162978. eCollection 2016.

13.

Gene therapy for metachromatic leukodystrophy.

Rosenberg JB, Kaminsky SM, Aubourg P, Crystal RG, Sondhi D.

J Neurosci Res. 2016 Nov;94(11):1169-79. doi: 10.1002/jnr.23792. Review.

14.

Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy.

Pagovich OE, Wang B, Chiuchiolo MJ, Kaminsky SM, Sondhi D, Jose CL, Price CC, Brooks SF, Mezey JG, Crystal RG.

J Allergy Clin Immunol. 2016 Dec;138(6):1652-1662.e7. doi: 10.1016/j.jaci.2016.03.053. Epub 2016 Jun 29.

PMID:
27372563
15.

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models.

Zhao L, Gottesdiener AJ, Parmar M, Li M, Kaminsky SM, Chiuchiolo MJ, Sondhi D, Sullivan PM, Holtzman DM, Crystal RG, Paul SM.

Neurobiol Aging. 2016 Aug;44:159-172. doi: 10.1016/j.neurobiolaging.2016.04.020. Epub 2016 Apr 30.

PMID:
27318144
16.

Brain Region-Specific Degeneration with Disease Progression in Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease).

Dyke JP, Sondhi D, Voss HU, Yohay K, Hollmann C, Mancenido D, Kaminsky SM, Heier LA, Rudser KD, Kosofsky B, Casey BJ, Crystal RG, Ballon D.

AJNR Am J Neuroradiol. 2016 Jun;37(6):1160-9. doi: 10.3174/ajnr.A4669. Epub 2016 Jan 28.

17.

Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.

Zerah M, Piguet F, Colle MA, Raoul S, Deschamps JY, Deniaud J, Gautier B, Toulgoat F, Bieche I, Laurendeau I, Sondhi D, Souweidane MM, Cartier-Lacave N, Moullier P, Crystal RG, Roujeau T, Sevin C, Aubourg P.

Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24. doi: 10.1089/humc.2014.139. Epub 2015 Apr 28.

PMID:
25758611
18.

Genetic modification of neurons to express bevacizumab for local anti-angiogenesis treatment of glioblastoma.

Hicks MJ, Funato K, Wang L, Aronowitz E, Dyke JP, Ballon DJ, Havlicek DF, Frenk EZ, De BP, Chiuchiolo MJ, Sondhi D, Hackett NR, Kaminsky SM, Tabar V, Crystal RG.

Cancer Gene Ther. 2015 Jan;22(1):1-8. doi: 10.1038/cgt.2014.58. Epub 2014 Dec 12.

19.

Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption.

Foley CP, Rubin DG, Santillan A, Sondhi D, Dyke JP, Crystal RG, Gobin YP, Ballon DJ.

J Control Release. 2014 Dec 28;196:71-78. doi: 10.1016/j.jconrel.2014.09.018. Epub 2014 Sep 28.

20.

Phase I/II study of intrapleural administration of a serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-antitrypsin cDNA to individuals with α1-antitrypsin deficiency.

Chiuchiolo MJ, Kaminsky SM, Sondhi D, Mancenido D, Hollmann C, Crystal RG.

Hum Gene Ther Clin Dev. 2014 Sep;25(3):112-33. doi: 10.1089/humc.2014.2513. No abstract available.

PMID:
25238276
21.

Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates.

Rosenberg JB, Sondhi D, Rubin DG, Monette S, Chen A, Cram S, De BP, Kaminsky SM, Sevin C, Aubourg P, Crystal RG.

Hum Gene Ther Clin Dev. 2014 Sep;25(3):164-77. doi: 10.1089/humc.2013.239. Epub 2014 Aug 21.

22.

Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.

Sondhi D, Scott EC, Chen A, Hackett NR, Wong AM, Kubiak A, Nelvagal HR, Pearse Y, Cotman SL, Cooper JD, Crystal RG.

Hum Gene Ther. 2014 Mar;25(3):223-39. doi: 10.1089/hum.2012.253. Epub 2014 Mar 4.

23.

Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway.

Lojewski X, Staropoli JF, Biswas-Legrand S, Simas AM, Haliw L, Selig MK, Coppel SH, Goss KA, Petcherski A, Chandrachud U, Sheridan SD, Lucente D, Sims KB, Gusella JF, Sondhi D, Crystal RG, Reinhardt P, Sterneckert J, Schöler H, Haggarty SJ, Storch A, Hermann A, Cotman SL.

Hum Mol Genet. 2014 Apr 15;23(8):2005-22. doi: 10.1093/hmg/ddt596. Epub 2013 Nov 23.

24.

Cannulation of the internal carotid artery in mice: a novel technique for intra-arterial delivery of therapeutics.

Santillan A, Rubin DG, Foley CP, Sondhi D, Crystal RG, Gobin YP, Ballon DJ.

J Neurosci Methods. 2014 Jan 30;222:106-10. doi: 10.1016/j.jneumeth.2013.11.008. Epub 2013 Nov 20.

PMID:
24269174
25.

Intrapleural administration of an AAVrh.10 vector coding for human α1-antitrypsin for the treatment of α1-antitrypsin deficiency.

Chiuchiolo MJ, Kaminsky SM, Sondhi D, Hackett NR, Rosenberg JB, Frenk EZ, Hwang Y, Van de Graaf BG, Hutt JA, Wang G, Benson J, Crystal RG.

Hum Gene Ther Clin Dev. 2013 Dec;24(4):161-73. doi: 10.1089/humc.2013.168.

PMID:
24191907
26.

Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease.

Kaminsky SM, Rosengart TK, Rosenberg J, Chiuchiolo MJ, Van de Graaf B, Sondhi D, Crystal RG.

Hum Gene Ther. 2013 Nov;24(11):948-63. doi: 10.1089/hum.2013.2516. Review.

PMID:
24164242
27.

Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function.

Orlin A, Sondhi D, Witmer MT, Wessel MM, Mezey JG, Kaminsky SM, Hackett NR, Yohay K, Kosofsky B, Souweidane MM, Kaplitt MG, D'Amico DJ, Crystal RG, Kiss S.

PLoS One. 2013 Aug 28;8(8):e73128. doi: 10.1371/journal.pone.0073128. eCollection 2013.

28.

Safety of direct cardiac administration of AdVEGF-All6A+, a replication-deficient adenovirus vector cDNA/genomic hybrid expressing all three major isoforms of human vascular endothelial growth factor, to the ischemic myocardium of rats.

Kaminsky SM, Quach L, Chen S, Pierre-Destine L, Van de Graaf B, Monette S, Rosenberg JB, De BP, Sondhi D, Hackett NR, Mezey JG, Rosengart TK, Crystal RG.

Hum Gene Ther Clin Dev. 2013 Mar;24(1):38-46. doi: 10.1089/humc.2013.054.

PMID:
23692382
29.

Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity.

De BP, Pagovich OE, Hicks MJ, Rosenberg JB, Moreno AY, Janda KD, Koob GF, Worgall S, Kaminsky SM, Sondhi D, Crystal RG.

Hum Gene Ther. 2013 Jan;24(1):58-66. doi: 10.1089/hum.2012.163. Epub 2012 Dec 21.

30.

Long-term expression and safety of administration of AAVrh.10hCLN2 to the brain of rats and nonhuman primates for the treatment of late infantile neuronal ceroid lipofuscinosis.

Sondhi D, Johnson L, Purpura K, Monette S, Souweidane MM, Kaplitt MG, Kosofsky B, Yohay K, Ballon D, Dyke J, Kaminksy SM, Hackett NR, Crystal RG.

Hum Gene Ther Methods. 2012 Oct;23(5):324-35. doi: 10.1089/hgtb.2012.120. Epub 2012 Nov 6.

31.

Assessment of disease severity in late infantile neuronal ceroid lipofuscinosis using multiparametric MR imaging.

Dyke JP, Sondhi D, Voss HU, Shungu DC, Mao X, Yohay K, Worgall S, Hackett NR, Hollmann C, Yeotsas ME, Jeong AL, Van de Graaf B, Cao I, Kaminsky SM, Heier LA, Rudser KD, Souweidane MM, Kaplitt MG, Kosofsky B, Crystal RG, Ballon D.

AJNR Am J Neuroradiol. 2013 Apr;34(4):884-9. doi: 10.3174/ajnr.A3297. Epub 2012 Oct 4.

32.

Novel therapies, high-risk pediatric research, and the prospect of benefit: learning from the ethical disagreements.

de Melo-Martín I, Sondhi D, Crystal RG.

Mol Ther. 2012 Jun;20(6):1095-102. doi: 10.1038/mt.2012.90. No abstract available.

33.

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice.

Piguet F, Sondhi D, Piraud M, Fouquet F, Hackett NR, Ahouansou O, Vanier MT, Bieche I, Aubourg P, Crystal RG, Cartier N, Sevin C.

Hum Gene Ther. 2012 Aug;23(8):903-14. doi: 10.1089/hum.2012.015. Epub 2012 Jul 23.

34.

When ethics constrains clinical research: trial design of control arms in "greater than minimal risk" pediatric trials.

de Melo-Martín I, Sondhi D, Crystal RG.

Hum Gene Ther. 2011 Sep;22(9):1121-7. doi: 10.1089/hum.2010.230. Epub 2011 May 19.

35.

Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations.

Souweidane MM, Fraser JF, Arkin LM, Sondhi D, Hackett NR, Kaminsky SM, Heier L, Kosofsky BE, Worgall S, Crystal RG, Kaplitt MG.

J Neurosurg Pediatr. 2010 Aug;6(2):115-22. doi: 10.3171/2010.4.PEDS09507.

36.

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis.

Sondhi D, Peterson DA, Edelstein AM, del Fierro K, Hackett NR, Crystal RG.

Exp Neurol. 2008 Sep;213(1):18-27. doi: 10.1016/j.expneurol.2008.04.022. Epub 2008 Apr 30.

37.

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

Worgall S, Sondhi D, Hackett NR, Kosofsky B, Kekatpure MV, Neyzi N, Dyke JP, Ballon D, Heier L, Greenwald BM, Christos P, Mazumdar M, Souweidane MM, Kaplitt MG, Crystal RG.

Hum Gene Ther. 2008 May;19(5):463-74. doi: 10.1089/hum.2008.022.

PMID:
18473686
38.

Assessing disease severity in late infantile neuronal ceroid lipofuscinosis using quantitative MR diffusion-weighted imaging.

Dyke JP, Voss HU, Sondhi D, Hackett NR, Worgall S, Heier LA, Kosofsky BE, Uluğ AM, Shungu DC, Mao X, Crystal RG, Ballon D.

AJNR Am J Neuroradiol. 2007 Aug;28(7):1232-6.

39.

Neurological deterioration in late infantile neuronal ceroid lipofuscinosis.

Worgall S, Kekatpure MV, Heier L, Ballon D, Dyke JP, Shungu D, Mao X, Kosofsky B, Kaplitt MG, Souweidane MM, Sondhi D, Hackett NR, Hollmann C, Crystal RG.

Neurology. 2007 Aug 7;69(6):521-35.

PMID:
17679671
40.

Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

Sondhi D, Hackett NR, Peterson DA, Stratton J, Baad M, Travis KM, Wilson JM, Crystal RG.

Mol Ther. 2007 Mar;15(3):481-91. Epub 2006 Dec 19.

41.

Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis.

Passini MA, Dodge JC, Bu J, Yang W, Zhao Q, Sondhi D, Hackett NR, Kaminsky SM, Mao Q, Shihabuddin LS, Cheng SH, Sleat DE, Stewart GR, Davidson BL, Lobel P, Crystal RG.

J Neurosci. 2006 Feb 1;26(5):1334-42.

42.

Safety of direct administration of AAV2(CU)hCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates.

Hackett NR, Redmond DE, Sondhi D, Giannaris EL, Vassallo E, Stratton J, Qiu J, Kaminsky SM, Lesser ML, Fisch GS, Rouselle SD, Crystal RG.

Hum Gene Ther. 2005 Dec;16(12):1484-503.

PMID:
16390279
43.

Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders.

Arkin LM, Sondhi D, Worgall S, Suh LH, Hackett NR, Kaminsky SM, Hosain SA, Souweidane MM, Kaplitt MG, Dyke JP, Heier LA, Ballon DJ, Shungu DC, Wisniewski KE, Greenwald BM, Hollmann C, Crystal RG.

Hum Gene Ther. 2005 Sep;16(9):1028-36. Review.

PMID:
16149901
44.

AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL.

Sondhi D, Peterson DA, Giannaris EL, Sanders CT, Mendez BS, De B, Rostkowski AB, Blanchard B, Bjugstad K, Sladek JR Jr, Redmond DE Jr, Leopold PL, Kaminsky SM, Hackett NR, Crystal RG.

Gene Ther. 2005 Nov;12(22):1618-32.

PMID:
16052206
45.

Clinical protocol. Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis.

Crystal RG, Sondhi D, Hackett NR, Kaminsky SM, Worgall S, Stieg P, Souweidane M, Hosain S, Heier L, Ballon D, Dinner M, Wisniewski K, Kaplitt M, Greenwald BM, Howell JD, Strybing K, Dyke J, Voss H.

Hum Gene Ther. 2004 Nov;15(11):1131-54.

PMID:
15610613
47.

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.

Sondhi D, Hackett NR, Apblett RL, Kaminsky SM, Pergolizzi RG, Crystal RG.

Arch Neurol. 2001 Nov;58(11):1793-8. Review.

PMID:
11708986
48.

Castleman's disease and superior vena cava thrombi: a rare presentation and a review of the literature.

Dhingra H, Sondhi D, Fleischman J, Ayinla R, Chawla K, Rosner F.

Mt Sinai J Med. 2001 Nov;68(6):410-6. Review.

PMID:
11687872
49.

Wernicke's encephalopathy in a non-alcoholic man: case report and brief review.

Munir A, Hussain SA, Sondhi D, Ameh J, Rosner F.

Mt Sinai J Med. 2001 May;68(3):216-8.

PMID:
11373696
50.

Microscopic polyangiitis presenting as idiopathic pulmonary fibrosis: is anti-neutrophilic cytoplasmic antibody testing indicated?

Mansi IA, Opran A, Sondhi D, Ayinla R, Rosner F.

Am J Med Sci. 2001 Mar;321(3):201-2.

PMID:
11269798

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