445 related articles for article (PubMed ID: 33365208)
1. Advances in the Treatment of Neuronal Ceroid Lipofuscinosis.
Rosenberg JB; Chen A; Kaminsky SM; Crystal RG; Sondhi D
Expert Opin Orphan Drugs; 2019; 7(11):473-500. PubMed ID: 33365208
[TBL] [Abstract][Full Text] [Related]
2. Experimental Therapeutic Approaches for the Treatment of Retinal Pathology in Neuronal Ceroid Lipofuscinoses.
Bartsch U; Storch S
Front Neurol; 2022; 13():866983. PubMed ID: 35509995
[TBL] [Abstract][Full Text] [Related]
3. 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]
4. Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis.
Lu JY; Nelvagal HR; Wang L; Birnbaum SG; Cooper JD; Hofmann SL
Mol Genet Metab; 2015; 116(1-2):98-105. PubMed ID: 25982063
[TBL] [Abstract][Full Text] [Related]
5. Neuronal Ceroid Lipofuscinosis: Potential for Targeted Therapy.
Specchio N; Ferretti A; Trivisano M; Pietrafusa N; Pepi C; Calabrese C; Livadiotti S; Simonetti A; Rossi P; Curatolo P; Vigevano F
Drugs; 2021 Jan; 81(1):101-123. PubMed ID: 33242182
[TBL] [Abstract][Full Text] [Related]
6. Therapeutic approaches to the challenge of neuronal ceroid lipofuscinoses.
Kohan R; Cismondi IA; Oller-Ramirez AM; Guelbert N; Anzolini TV; Alonso G; Mole SE; de Kremer DR; de Halac NI
Curr Pharm Biotechnol; 2011 Jun; 12(6):867-83. PubMed ID: 21235444
[TBL] [Abstract][Full Text] [Related]
7. Mouse models of neuronal ceroid lipofuscinoses: useful pre-clinical tools to delineate disease pathophysiology and validate therapeutics.
Shacka JJ
Brain Res Bull; 2012 May; 88(1):43-57. PubMed ID: 22502604
[TBL] [Abstract][Full Text] [Related]
8. Neural stem cells for disease modeling and evaluation of therapeutics for infantile (CLN1/PPT1) and late infantile (CLN2/TPP1) neuronal ceroid lipofuscinoses.
Sima N; Li R; Huang W; Xu M; Beers J; Zou J; Titus S; Ottinger EA; Marugan JJ; Xie X; Zheng W
Orphanet J Rare Dis; 2018 Apr; 13(1):54. PubMed ID: 29631617
[TBL] [Abstract][Full Text] [Related]
9. Cln3-mutations underlying juvenile neuronal ceroid lipofuscinosis cause significantly reduced levels of Palmitoyl-protein thioesterases-1 (Ppt1)-protein and Ppt1-enzyme activity in the lysosome.
Appu AP; Bagh MB; Sadhukhan T; Mondal A; Casey S; Mukherjee AB
J Inherit Metab Dis; 2019 Sep; 42(5):944-954. PubMed ID: 31025705
[TBL] [Abstract][Full Text] [Related]
10. Cell biology of the NCL proteins: What they do and don't do.
Cárcel-Trullols J; Kovács AD; Pearce DA
Biochim Biophys Acta; 2015 Oct; 1852(10 Pt B):2242-55. PubMed ID: 25962910
[TBL] [Abstract][Full Text] [Related]
11. Pathogenesis and therapies for infantile neuronal ceroid lipofuscinosis (infantile CLN1 disease).
Hawkins-Salsbury JA; Cooper JD; Sands MS
Biochim Biophys Acta; 2013 Nov; 1832(11):1906-9. PubMed ID: 23747979
[TBL] [Abstract][Full Text] [Related]
12. The neuronal ceroid lipofuscinoses: mutations in different proteins result in similar disease.
Weimer JM; Kriscenski-Perry E; Elshatory Y; Pearce DA
Neuromolecular Med; 2002; 1(2):111-24. PubMed ID: 12025857
[TBL] [Abstract][Full Text] [Related]
13. Neuronal ceroid lipofuscinoses: classification and diagnosis.
Wisniewski KE; Kida E; Golabek AA; Kaczmarski W; Connell F; Zhong N
Adv Genet; 2001; 45():1-34. PubMed ID: 11332767
[TBL] [Abstract][Full Text] [Related]
14. Enzyme replacement therapy with recombinant pro-CTSD (cathepsin D) corrects defective proteolysis and autophagy in neuronal ceroid lipofuscinosis.
Marques ARA; Di Spiezio A; Thießen N; Schmidt L; Grötzinger J; Lüllmann-Rauch R; Damme M; Storck SE; Pietrzik CU; Fogh J; Bär J; Mikhaylova M; Glatzel M; Bassal M; Bartsch U; Saftig P
Autophagy; 2020 May; 16(5):811-825. PubMed ID: 31282275
[TBL] [Abstract][Full Text] [Related]
15. Using the social amoeba Dictyostelium to study the functions of proteins linked to neuronal ceroid lipofuscinosis.
Huber RJ
J Biomed Sci; 2016 Nov; 23(1):83. PubMed ID: 27881166
[TBL] [Abstract][Full Text] [Related]
16. Novel interactions of CLN5 support molecular networking between Neuronal Ceroid Lipofuscinosis proteins.
Lyly A; von Schantz C; Heine C; Schmiedt ML; Sipilä T; Jalanko A; Kyttälä A
BMC Cell Biol; 2009 Nov; 10():83. PubMed ID: 19941651
[TBL] [Abstract][Full Text] [Related]
17. Human pathology in NCL.
Anderson GW; Goebel HH; Simonati A
Biochim Biophys Acta; 2013 Nov; 1832(11):1807-26. PubMed ID: 23200925
[TBL] [Abstract][Full Text] [Related]
18. Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis.
Geraets RD; Koh Sy; Hastings ML; Kielian T; Pearce DA; Weimer JM
Orphanet J Rare Dis; 2016 Apr; 11():40. PubMed ID: 27083890
[TBL] [Abstract][Full Text] [Related]
19. Clinical and genetic characterization of neuronal ceroid lipofuscinoses (NCLs) in 29 Iranian patients: identification of 11 novel mutations.
Panjeshahi S; Karimzadeh P; Movafagh A; Ahmadabadi F; Rahimian E; Alijanpour S; Miryounesi M
Hum Genet; 2023 Aug; 142(8):1001-1016. PubMed ID: 37074398
[TBL] [Abstract][Full Text] [Related]
20.
; ; . PubMed ID:
[No Abstract] [Full Text] [Related]
[Next] [New Search]