BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

203 related articles for article (PubMed ID: 37955580)

  • 1. Intrauterine enzyme replacement therapies for lysosomal storage disorders: Current developments and promising future prospects.
    Herzeg A; Borges B; Lianoglou BR; Gonzalez-Velez J; Canepa E; Munar D; Young SP; Bali D; Gelb MH; Chakraborty P; Kishnani PS; Harmatz P; Cohen JL; MacKenzie TC
    Prenat Diagn; 2023 Dec; 43(13):1638-1649. PubMed ID: 37955580
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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]  

  • 4. Fetal therapies and trials for lysosomal storage diseases: a survey of attitudes of parents and patients.
    Schwab ME; Brown JEH; Lianoglou B; Jin C; Conroy PC; Gallagher RC; Harmatz P; MacKenzie TC
    Orphanet J Rare Dis; 2022 Jan; 17(1):25. PubMed ID: 35093147
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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]  

  • 6. Pulmonary involvement in selected lysosomal storage diseases and the impact of enzyme replacement therapy: A state-of-the art review.
    Jezela-Stanek A; Chorostowska-Wynimko J; Tylki-Szymańska A
    Clin Respir J; 2020 May; 14(5):422-429. PubMed ID: 31912638
    [TBL] [Abstract][Full Text] [Related]  

  • 7. Enzyme enhancement therapeutics for lysosomal storage diseases: Current status and perspective.
    Thomas R; Kermode AR
    Mol Genet Metab; 2019 Feb; 126(2):83-97. PubMed ID: 30528228
    [TBL] [Abstract][Full Text] [Related]  

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

  • 9. Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.
    Macauley SL
    Pediatr Endocrinol Rev; 2016 Jun; 13 Suppl 1(Suppl 1):639-48. PubMed ID: 27491211
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Stem Cell Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges.
    Köse S; Aerts-Kaya F; Uçkan Çetinkaya D; Korkusuz P
    Adv Exp Med Biol; 2021; 1347():135-162. PubMed ID: 33977438
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Emerging therapies for neuropathic lysosomal storage disorders.
    Kelly JM; Bradbury A; Martin DR; Byrne ME
    Prog Neurobiol; 2017 May; 152():166-180. PubMed ID: 27725193
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII.
    Nguyen QH; Witt RG; Wang B; Eikani C; Shea J; Smith LK; Boyle G; Cadaoas J; Sper R; MacKenzie JD; Villeda S; MacKenzie TC
    Sci Transl Med; 2020 Feb; 12(532):. PubMed ID: 32102934
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Treatment of lysosomal storage diseases: recent patents and future strategies.
    Ortolano S; Viéitez I; Navarro C; Spuch C
    Recent Pat Endocr Metab Immune Drug Discov; 2014 Jan; 8(1):9-25. PubMed ID: 24433521
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Oral small molecule therapy for lysosomal storage diseases.
    Weinreb NJ
    Pediatr Endocrinol Rev; 2013 Nov; 11 Suppl 1():77-90. PubMed ID: 24380126
    [TBL] [Abstract][Full Text] [Related]  

  • 15. New Advanced Strategies for the Treatment of Lysosomal Diseases Affecting the Central Nervous System.
    Gigliobianco MR; Di Martino P; Deng S; Casadidio C; Censi R
    Curr Pharm Des; 2019; 25(17):1933-1950. PubMed ID: 31566121
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Enzyme Replacement Therapy: A Review and Its Role in Treating Lysosomal Storage Diseases.
    Li M
    Pediatr Ann; 2018 May; 47(5):e191-e197. PubMed ID: 29750286
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Evolutionary redesign of the lysosomal enzyme arylsulfatase A increases efficacy of enzyme replacement therapy for metachromatic leukodystrophy.
    Simonis H; Yaghootfam C; Sylvester M; Gieselmann V; Matzner U
    Hum Mol Genet; 2019 Jun; 28(11):1810-1821. PubMed ID: 30657900
    [TBL] [Abstract][Full Text] [Related]  

  • 18. 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]  

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

  • 20. Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.
    Solomon M; Muro S
    Adv Drug Deliv Rev; 2017 Sep; 118():109-134. PubMed ID: 28502768
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 11.