These tools will no longer be maintained as of December 31, 2024. Archived website can be found here. PubMed4Hh GitHub repository can be found here. Contact NLM Customer Service if you have questions.


BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

360 related articles for article (PubMed ID: 34944420)

  • 1. Therapeutic Approaches in Lysosomal Storage Diseases.
    Fernández-Pereira C; San Millán-Tejado B; Gallardo-Gómez M; Pérez-Márquez T; Alves-Villar M; Melcón-Crespo C; Fernández-Martín J; Ortolano S
    Biomolecules; 2021 Nov; 11(12):. PubMed ID: 34944420
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Lysosomal storage diseases: from pathophysiology to therapy.
    Parenti G; Andria G; Ballabio A
    Annu Rev Med; 2015; 66():471-86. PubMed ID: 25587658
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Enzyme replacement therapy and beyond-in memoriam Roscoe O. Brady, M.D. (1923-2016).
    Ries M
    J Inherit Metab Dis; 2017 May; 40(3):343-356. PubMed ID: 28314976
    [TBL] [Abstract][Full Text] [Related]  

  • 4. Biomarkers for gene therapy clinical trials of lysosomal storage disorders.
    Rossi A; Malvagia S; la Marca G; Parenti G; Brunetti-Pierri N
    Mol Ther; 2024 Sep; 32(9):2930-2938. PubMed ID: 38850023
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Delivering Hematopoietic Stem Cell Gene Therapy Treatments for Neurological Lysosomal Diseases.
    Holley RJ; Wood SR; Bigger BW
    ACS Chem Neurosci; 2019 Jan; 10(1):18-20. PubMed ID: 30136572
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Gene therapy approaches for lysosomal storage disease: next-generation treatment.
    Byrne BJ; Falk DJ; Clément N; Mah CS
    Hum Gene Ther; 2012 Aug; 23(8):808-15. PubMed ID: 22794786
    [TBL] [Abstract][Full Text] [Related]  

  • 7.
    Poletto E; Silva AO; Weinlich R; Martin PKM; Torres DC; Giugliani R; Baldo G
    Expert Opin Biol Ther; 2023 Apr; 23(4):353-364. PubMed ID: 36920351
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.
    Massaro G; Geard AF; Liu W; Coombe-Tennant O; Waddington SN; Baruteau J; Gissen P; Rahim AA
    Biomolecules; 2021 Apr; 11(4):. PubMed ID: 33924076
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Treatments for lysosomal storage disorders.
    Lachmann R
    Biochem Soc Trans; 2010 Dec; 38(6):1465-8. PubMed ID: 21118108
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Treatment strategies for lysosomal storage disorders.
    Beck M
    Dev Med Child Neurol; 2018 Jan; 60(1):13-18. PubMed ID: 29090451
    [TBL] [Abstract][Full Text] [Related]  

  • 11. New strategies for the treatment of lysosomal storage diseases (review).
    Parenti G; Pignata C; Vajro P; Salerno M
    Int J Mol Med; 2013 Jan; 31(1):11-20. PubMed ID: 23165354
    [TBL] [Abstract][Full Text] [Related]  

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

  • 13. Recent developments in therapeutic approaches for lysosomal storage diseases.
    Urbanelli L; Magini A; Polchi A; Polidoro M; Emiliani C
    Recent Pat CNS Drug Discov; 2011 Jan; 6(1):1-19. PubMed ID: 21073432
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Gene therapy for lysosomal storage diseases and peroxisomal diseases.
    Ohashi T
    J Hum Genet; 2019 Feb; 64(2):139-143. PubMed ID: 30498239
    [TBL] [Abstract][Full Text] [Related]  

  • 15. 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; 40(4):543-554. PubMed ID: 28560469
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Eighth International Symposium on Lysosomal Storage Diseases.
    Mehta AB; Germain DP; Martin R
    Int J Clin Pharmacol Ther; 2009; 47 Suppl 1():S1-3. PubMed ID: 20040305
    [No Abstract]   [Full Text] [Related]  

  • 17. Recent advances in treatment approaches of lysosomal storage diseases (LSDs).
    Rozenfeld PA
    Curr Pharm Biotechnol; 2011 Jun; 12(6):853. PubMed ID: 21506916
    [No Abstract]   [Full Text] [Related]  

  • 18. [Therapy of lysosomal storage diseases: update and perspectives].
    Lara-Aguilar RA; Juárez-Vázquez CI; Medina-Lozano C
    Rev Invest Clin; 2011; 63(6):651-8. PubMed ID: 23650678
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Lysosomal Storage Disorders: Molecular Basis and Therapeutic Approaches.
    Moro E
    Biomolecules; 2021 Jun; 11(7):. PubMed ID: 34208892
    [TBL] [Abstract][Full Text] [Related]  

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

    [Next]    [New Search]
    of 18.