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Journal Abstract Search

514 related items for PubMed ID: 24433521

  • 21. Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders.
    Del Grosso A, Parlanti G, Mezzena R, Cecchini M.
    Adv Drug Deliv Rev; 2022 Sep; 188():114464. PubMed ID: 35878795
    [Abstract] [Full Text] [Related]

  • 22. Gene therapy for lysosomal storage diseases (LSDs) in large animal models.
    Haskins M.
    ILAR J; 2009 Sep; 50(2):112-21. PubMed ID: 19293456
    [Abstract] [Full Text] [Related]

  • 23. [Enzyme replacement therapy of lysosomal storage diseases].
    Germain DP, Boucly C, Carlier RY, Caudron E, Charlier P, Colas F, Jabbour F, Martinez V, Mokhtari S, Orlikowski D, Pellegrini N, Perronne C, Prigent H, Rubinsztajn R, Benistan K.
    Rev Med Interne; 2010 Dec; 31 Suppl 2():S279-91. PubMed ID: 21211680
    [Abstract] [Full Text] [Related]

  • 24. Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier.
    Critchley BJ, Gaspar HB, Benedetti S.
    Mol Ther; 2023 Mar 01; 31(3):657-675. PubMed ID: 36457248
    [Abstract] [Full Text] [Related]

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  • 29. Advances in therapies for neurological lysosomal storage disorders.
    Ellison S, Parker H, Bigger B.
    J Inherit Metab Dis; 2023 Sep 01; 46(5):874-905. PubMed ID: 37078180
    [Abstract] [Full Text] [Related]

  • 30. Therapeutic Role of Pharmacological Chaperones in Lysosomal Storage Disorders: A Review of the Evidence and Informed Approach to Reclassification.
    Keyzor I, Shohet S, Castelli J, Sitaraman S, Veleva-Rotse B, Weimer JM, Fox B, Willer T, Tuske S, Crathorne L, Belzar KJ.
    Biomolecules; 2023 Aug 07; 13(8):. PubMed ID: 37627292
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  • 31. Lysosomal storage diseases: Stem cell-based cell- and gene-therapy.
    Kim SU.
    Cell Transplant; 2014 May 21. PubMed ID: 24853878
    [Abstract] [Full Text] [Related]

  • 32. CNS-directed gene therapy for lysosomal storage diseases.
    Sands MS, Haskins ME.
    Acta Paediatr; 2008 Apr 21; 97(457):22-7. PubMed ID: 18339183
    [Abstract] [Full Text] [Related]

  • 33. Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications.
    Biffi A.
    Mol Ther; 2017 May 03; 25(5):1155-1162. PubMed ID: 28389320
    [Abstract] [Full Text] [Related]

  • 34. Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I.
    Penati R, Fumagalli F, Calbi V, Bernardo ME, Aiuti A.
    J Inherit Metab Dis; 2017 Jul 03; 40(4):543-554. PubMed ID: 28560469
    [Abstract] [Full Text] [Related]

  • 35. New strategies for enzyme replacement therapy for lysosomal storage diseases.
    Grubb JH, Vogler C, Sly WS.
    Rejuvenation Res; 2010 Jul 03; 13(2-3):229-36. PubMed ID: 20345279
    [Abstract] [Full Text] [Related]

  • 36. Current state of the management of LSDs.
    Germain DP, Aggio M.
    Int J Clin Pharmacol Ther; 2009 Jul 03; 47 Suppl 1():S61-2. PubMed ID: 20040313
    [No Abstract] [Full Text] [Related]

  • 37. Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease.
    Alliegro M, Ferla R, Nusco E, De Leonibus C, Settembre C, Auricchio A.
    Mol Ther; 2016 Dec 03; 24(12):2054-2063. PubMed ID: 27658524
    [Abstract] [Full Text] [Related]

  • 38. Perspectives from B cell immunology: fact and fancy.
    Hunt SV.
    Int J Clin Pharmacol Ther; 2009 Dec 03; 47 Suppl 1():S86-99. PubMed ID: 20040318
    [Abstract] [Full Text] [Related]

  • 39. Novel therapies and future perspectives.
    Scarpa M, Frustaci A.
    Int J Clin Pharmacol Ther; 2009 Dec 03; 47 Suppl 1():S109-10. PubMed ID: 20040320
    [No Abstract] [Full Text] [Related]

  • 40. Genetics and Gene Therapy of Anderson-Fabry Disease.
    Simonetta I, Tuttolomondo A, Di Chiara T, Miceli S, Vogiatzis D, Corpora F, Pinto A.
    Curr Gene Ther; 2018 Dec 03; 18(2):96-106. PubMed ID: 29618309
    [Abstract] [Full Text] [Related]

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