BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

476 related articles for article (PubMed ID: 21811247)

  • 1. Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders.
    Bevan AK; Duque S; Foust KD; Morales PR; Braun L; Schmelzer L; Chan CM; McCrate M; Chicoine LG; Coley BD; Porensky PN; Kolb SJ; Mendell JR; Burghes AH; Kaspar BK
    Mol Ther; 2011 Nov; 19(11):1971-80. PubMed ID: 21811247
    [TBL] [Abstract][Full Text] [Related]  

  • 2. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates.
    Meyer K; Ferraiuolo L; Schmelzer L; Braun L; McGovern V; Likhite S; Michels O; Govoni A; Fitzgerald J; Morales P; Foust KD; Mendell JR; Burghes AH; Kaspar BK
    Mol Ther; 2015 Mar; 23(3):477-87. PubMed ID: 25358252
    [TBL] [Abstract][Full Text] [Related]  

  • 3. Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice.
    Benkhelifa-Ziyyat S; Besse A; Roda M; Duque S; Astord S; Carcenac R; Marais T; Barkats M
    Mol Ther; 2013 Feb; 21(2):282-90. PubMed ID: 23295949
    [TBL] [Abstract][Full Text] [Related]  

  • 4. AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice.
    Besse A; Astord S; Marais T; Roda M; Giroux B; Lejeune FX; Relaix F; Smeriglio P; Barkats M; Biferi MG
    Mol Ther; 2020 Aug; 28(8):1887-1901. PubMed ID: 32470325
    [TBL] [Abstract][Full Text] [Related]  

  • 5. The advent of AAV9 expands applications for brain and spinal cord gene delivery.
    Dayton RD; Wang DB; Klein RL
    Expert Opin Biol Ther; 2012 Jun; 12(6):757-66. PubMed ID: 22519910
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Efficient CNS targeting in adult mice by intrathecal infusion of single-stranded AAV9-GFP for gene therapy of neurological disorders.
    Bey K; Ciron C; Dubreil L; Deniaud J; Ledevin M; Cristini J; Blouin V; Aubourg P; Colle MA
    Gene Ther; 2017 May; 24(5):325-332. PubMed ID: 28425480
    [TBL] [Abstract][Full Text] [Related]  

  • 7. AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice.
    Liguore WA; Domire JS; Button D; Wang Y; Dufour BD; Srinivasan S; McBride JL
    Mol Ther; 2019 Nov; 27(11):2018-2037. PubMed ID: 31420242
    [TBL] [Abstract][Full Text] [Related]  

  • 8. Translational fidelity of intrathecal delivery of self-complementary AAV9-survival motor neuron 1 for spinal muscular atrophy.
    Passini MA; Bu J; Richards AM; Treleaven CM; Sullivan JA; O'Riordan CR; Scaria A; Kells AP; Samaranch L; San Sebastian W; Federici T; Fiandaca MS; Boulis NM; Bankiewicz KS; Shihabuddin LS; Cheng SH
    Hum Gene Ther; 2014 Jul; 25(7):619-30. PubMed ID: 24617515
    [TBL] [Abstract][Full Text] [Related]  

  • 9. Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates.
    Gray SJ; Nagabhushan Kalburgi S; McCown TJ; Jude Samulski R
    Gene Ther; 2013 Apr; 20(4):450-9. PubMed ID: 23303281
    [TBL] [Abstract][Full Text] [Related]  

  • 10. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies.
    Bailey RM; Rozenberg A; Gray SJ
    Brain Res; 2020 Jul; 1739():146832. PubMed ID: 32289279
    [TBL] [Abstract][Full Text] [Related]  

  • 11. Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice.
    Rashnonejad A; Amini Chermahini G; Gündüz C; Onay H; Aykut A; Durmaz B; Baka M; Su Q; Gao G; Özkınay F
    Mol Ther; 2019 Dec; 27(12):2123-2133. PubMed ID: 31543414
    [TBL] [Abstract][Full Text] [Related]  

  • 12. Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats.
    Bucher T; Dubreil L; Colle MA; Maquigneau M; Deniaud J; Ledevin M; Moullier P; Joussemet B
    Gene Ther; 2014 May; 21(5):522-8. PubMed ID: 24572783
    [TBL] [Abstract][Full Text] [Related]  

  • 13. Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy.
    Reilly A; Deguise MO; Beauvais A; Yaworski R; Thebault S; Tessier DR; Tabard-Cossa V; Hensel N; Schneider BL; Kothary R
    Gene Ther; 2022 Sep; 29(9):544-554. PubMed ID: 35462564
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.
    Foust KD; Nurre E; Montgomery CL; Hernandez A; Chan CM; Kaspar BK
    Nat Biotechnol; 2009 Jan; 27(1):59-65. PubMed ID: 19098898
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Intracerebroventricular injection of adeno-associated virus 6 and 9 vectors for cell type-specific transgene expression in the spinal cord.
    Dirren E; Towne CL; Setola V; Redmond DE; Schneider BL; Aebischer P
    Hum Gene Ther; 2014 Feb; 25(2):109-20. PubMed ID: 24191919
    [TBL] [Abstract][Full Text] [Related]  

  • 16. The AAV9 Variant Capsid AAV-F Mediates Widespread Transgene Expression in Nonhuman Primate Spinal Cord After Intrathecal Administration.
    Beharry A; Gong Y; Kim JC; Hanlon KS; Nammour J; Hieber K; Eichler F; Cheng M; Stemmer-Rachamimov A; Stankovic KM; Welling DB; Ng C; Maguire CA
    Hum Gene Ther; 2022 Jan; 33(1-2):61-75. PubMed ID: 34128391
    [TBL] [Abstract][Full Text] [Related]  

  • 17. Strong cortical and spinal cord transduction after AAV7 and AAV9 delivery into the cerebrospinal fluid of nonhuman primates.
    Samaranch L; Salegio EA; San Sebastian W; Kells AP; Bringas JR; Forsayeth J; Bankiewicz KS
    Hum Gene Ther; 2013 May; 24(5):526-32. PubMed ID: 23517473
    [TBL] [Abstract][Full Text] [Related]  

  • 18. Premature delivery in the domestic sow in response to in utero delivery of AAV9 to fetal piglets.
    Rich KA; Wier CG; Russo J; Kong L; Heilman PL; Reynolds A; Knapp A; Pino MG; Keckley E; Mattox L; Malbrue RA; Sumner CJ; Buhimschi C; Kolb SJ
    Gene Ther; 2022 Sep; 29(9):513-519. PubMed ID: 34803165
    [TBL] [Abstract][Full Text] [Related]  

  • 19. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs.
    Federici T; Taub JS; Baum GR; Gray SJ; Grieger JC; Matthews KA; Handy CR; Passini MA; Samulski RJ; Boulis NM
    Gene Ther; 2012 Aug; 19(8):852-9. PubMed ID: 21918551
    [TBL] [Abstract][Full Text] [Related]  

  • 20. Intravenous administration of the adeno-associated virus-PHP.B capsid fails to upregulate transduction efficiency in the marmoset brain.
    Matsuzaki Y; Konno A; Mochizuki R; Shinohara Y; Nitta K; Okada Y; Hirai H
    Neurosci Lett; 2018 Feb; 665():182-188. PubMed ID: 29175632
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 24.