BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

252 related articles for article (PubMed ID: 21291356)

  • 1. Therapies for neurological disease in the mucopolysaccharidoses.
    Anson DS; McIntyre C; Byers S
    Curr Gene Ther; 2011 Apr; 11(2):132-43. PubMed ID: 21291356
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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]  

  • 3. Treatment of brain disease in the mucopolysaccharidoses.
    Scarpa M; Orchard PJ; Schulz A; Dickson PI; Haskins ME; Escolar ML; Giugliani R
    Mol Genet Metab; 2017 Dec; 122S():25-34. PubMed ID: 29153844
    [TBL] [Abstract][Full Text] [Related]  

  • 4. New treatments for the mucopolysaccharidoses: from pathophysiology to therapy.
    Fecarotta S; Gasperini S; Parenti G
    Ital J Pediatr; 2018 Nov; 44(Suppl 2):124. PubMed ID: 30442204
    [TBL] [Abstract][Full Text] [Related]  

  • 5. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.
    Safary A; Akbarzadeh Khiavi M; Omidi Y; Rafi MA
    Cell Mol Life Sci; 2019 Sep; 76(17):3363-3381. PubMed ID: 31101939
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses.
    Pan D
    Curr Pharm Biotechnol; 2011 Jun; 12(6):884-96. PubMed ID: 21235445
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS.
    Giugliani R; Vairo F; Kubaski F; Poswar F; Riegel M; Baldo G; Saute JA
    Lancet Child Adolesc Health; 2018 Jan; 2(1):56-68. PubMed ID: 30169196
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Current and potential therapeutic strategies for mucopolysaccharidoses.
    Noh H; Lee JI
    J Clin Pharm Ther; 2014 Jun; 39(3):215-24. PubMed ID: 24612142
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.
    Lau AA; Hemsley KM
    J Mol Med (Berl); 2017 Oct; 95(10):1043-1052. PubMed ID: 28660346
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Gene therapy for neurologic manifestations of mucopolysaccharidoses.
    Wolf DA; Banerjee S; Hackett PB; Whitley CB; McIvor RS; Low WC
    Expert Opin Drug Deliv; 2015 Feb; 12(2):283-96. PubMed ID: 25510418
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future.
    Chen HH; Sawamoto K; Mason RW; Kobayashi H; Yamaguchi S; Suzuki Y; Orii K; Orii T; Tomatsu S
    J Hum Genet; 2019 Nov; 64(11):1153-1171. PubMed ID: 31455839
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Large animal models contribute to the development of therapies for central and peripheral nervous system dysfunction in patients with lysosomal storage diseases.
    Gurda BL; Vite CH
    Hum Mol Genet; 2019 Oct; 28(R1):R119-R131. PubMed ID: 31384936
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Nanotechnology applied to treatment of mucopolysaccharidoses.
    Schuh RS; Baldo G; Teixeira HF
    Expert Opin Drug Deliv; 2016 Dec; 13(12):1709-1718. PubMed ID: 27322551
    [TBL] [Abstract][Full Text] [Related]  

  • 14. [Treatment prospects of lysosomal storage disorders].
    Reismann P; Tulassay Z
    Orv Hetil; 2008 Jun; 149(25):1171-9. PubMed ID: 18547894
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Emerging drugs for the treatment of mucopolysaccharidoses.
    Giugliani R; Federhen A; Vairo F; Vanzella C; Pasqualim G; da Silva LM; Giugliani L; de Boer AP; de Souza CF; Matte U; Baldo G
    Expert Opin Emerg Drugs; 2016; 21(1):9-26. PubMed ID: 26751109
    [TBL] [Abstract][Full Text] [Related]  

  • 16. New prospects for the treatment of lysosomal storage diseases.
    Schiffmann R; Brady RO
    Drugs; 2002; 62(5):733-42. PubMed ID: 11929328
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Enzyme replacement therapy and substrate reduction therapy in lysosomal storage disorders with neurological expression.
    Valayannopoulos V
    Handb Clin Neurol; 2013; 113():1851-7. PubMed ID: 23622408
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Gene editing strategies to treat lysosomal disorders: The example of mucopolysaccharidoses.
    Fachel FNS; Frâncio L; Poletto É; Schuh RS; Teixeira HF; Giugliani R; Baldo G; Matte U
    Adv Drug Deliv Rev; 2022 Dec; 191():114616. PubMed ID: 36356930
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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; 31(3):657-675. PubMed ID: 36457248
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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]  

    [Next]    [New Search]
    of 13.