BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

163 related articles for article (PubMed ID: 37624739)

  • 1. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.
    De BP; Rosenberg JB; Selvan N; Wilson I; Yusufzai N; Greco A; Kaminsky SM; Heier LA; Ricart Arbona RJ; Miranda IC; Monette S; Nair A; Khanna R; Crystal RG; Sondhi D
    Hum Gene Ther; 2023 Sep; 34(17-18):905-916. PubMed ID: 37624739
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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. PubMed ID: 23131032
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing
    Sondhi D; Kaminsky SM; Hackett NR; Pagovich OE; Rosenberg JB; De BP; Chen A; Van de Graaf B; Mezey JG; Mammen GW; Mancenido D; Xu F; Kosofsky B; Yohay K; Worgall S; Kaner RJ; Souwedaine M; Greenwald BM; Kaplitt M; Dyke JP; Ballon DJ; Heier LA; Kiss S; Crystal RG
    Sci Transl Med; 2020 Dec; 12(572):. PubMed ID: 33268510
    [TBL] [Abstract][Full Text] [Related]  

  • 4. 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. PubMed ID: 18639872
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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. PubMed ID: 16390279
    [TBL] [Abstract][Full Text] [Related]  

  • 6. 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. PubMed ID: 17180118
    [TBL] [Abstract][Full Text] [Related]  

  • 7. 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; Redmond DE; Leopold PL; Kaminsky SM; Hackett NR; Crystal RG
    Gene Ther; 2005 Nov; 12(22):1618-32. PubMed ID: 16052206
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy.
    Rosenberg JB; Chen A; De BP; Dyke JP; Ballon DJ; Monette S; Ricart Arbona RJ; Kaminsky SM; Crystal RG; Sondhi D
    Hum Gene Ther; 2021 Jun; 32(11-12):563-580. PubMed ID: 33380277
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis.
    Vuillemenot BR; Kennedy D; Cooper JD; Wong AM; Sri S; Doeleman T; Katz ML; Coates JR; Johnson GC; Reed RP; Adams EL; Butt MT; Musson DG; Henshaw J; Keve S; Cahayag R; Tsuruda LS; O'Neill CA
    Mol Genet Metab; 2015 Feb; 114(2):281-93. PubMed ID: 25257657
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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. PubMed ID: 15610613
    [TBL] [Abstract][Full Text] [Related]  

  • 11. 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. PubMed ID: 24372003
    [TBL] [Abstract][Full Text] [Related]  

  • 12. CLN2 Disease (Classic Late Infantile Neuronal Ceroid Lipofuscinosis).
    Kohlschütter A; Schulz A
    Pediatr Endocrinol Rev; 2016 Jun; 13 Suppl 1():682-8. PubMed ID: 27491216
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease).
    Katz ML; Coates JR; Sibigtroth CM; Taylor JD; Carpentier M; Young WM; Wininger FA; Kennedy D; Vuillemenot BR; O'Neill CA
    J Neurosci Res; 2014 Nov; 92(11):1591-8. PubMed ID: 24938720
    [TBL] [Abstract][Full Text] [Related]  

  • 14. 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. PubMed ID: 25144894
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease.
    Whiting REH; Jensen CA; Pearce JW; Gillespie LE; Bristow DE; Katz ML
    Exp Eye Res; 2016 May; 146():276-282. PubMed ID: 27039708
    [TBL] [Abstract][Full Text] [Related]  

  • 16. A mouse model of classical late-infantile neuronal ceroid lipofuscinosis based on targeted disruption of the CLN2 gene results in a loss of tripeptidyl-peptidase I activity and progressive neurodegeneration.
    Sleat DE; Wiseman JA; El-Banna M; Kim KH; Mao Q; Price S; Macauley SL; Sidman RL; Shen MM; Zhao Q; Passini MA; Davidson BL; Stewart GR; Lobel P
    J Neurosci; 2004 Oct; 24(41):9117-26. PubMed ID: 15483130
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Intravitreal gene therapy preserves retinal function in a canine model of CLN2 neuronal ceroid lipofuscinosis.
    Kick GR; Whiting REH; Ota-Kuroki J; Castaner LJ; Morgan-Jack B; Sabol JC; Meiman EJ; Ortiz F; Katz ML
    Exp Eye Res; 2023 Jan; 226():109344. PubMed ID: 36509165
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression.
    Katz ML; Johnson GC; Leach SB; Williamson BG; Coates JR; Whiting REH; Vansteenkiste DP; Whitney MS
    Gene Ther; 2017 Apr; 24(4):215-223. PubMed ID: 28079862
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Multifocal retinopathy in Dachshunds with CLN2 neuronal ceroid lipofuscinosis.
    Whiting RE; Pearce JW; Castaner LJ; Jensen CA; Katz RJ; Gilliam DH; Katz ML
    Exp Eye Res; 2015 May; 134():123-32. PubMed ID: 25697710
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease): Expert recommendations for early detection and laboratory diagnosis.
    Fietz M; AlSayed M; Burke D; Cohen-Pfeffer J; Cooper JD; Dvořáková L; Giugliani R; Izzo E; Jahnová H; Lukacs Z; Mole SE; Noher de Halac I; Pearce DA; Poupetova H; Schulz A; Specchio N; Xin W; Miller N
    Mol Genet Metab; 2016 Sep; 119(1-2):160-7. PubMed ID: 27553878
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 9.