Adeno-Associated Virus-Based Gene Therapy for CNS Diseases

Cites (220)

Items 201–219 of 219

1. Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy
   Sung-Chul Jung, E. Park, E. Choi, C. Kim, S. Kim, D. Jin · 2010 · Molecules and Cells
2. Restoration of central nervous system α‐N‐acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno‐associated viral type 2 vector delivery
   Haiyan Fu, Julianne DiRosario, Kang Lu, Joseph Muenzer, Douglas M. McCarty · 2010 · The Journal of Gene Medicine
3. Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice
   Coy Don Heldermon, Elizabeth Y. Qin, Kevin K. Ohlemiller, Erik D. Herzog, Jeremy R. Glissen Brown, Carole A. Vogler, Wei Hou, John L. Orrock, Brett E. Crawford, Mark S. Sands · 2013 · Gene Therapy
4. Long-term correction of biochemical and neurological abnormalities in MLD mice model by neonatal systemic injection of an AAV serotype 9 vector
   Noriko Miyake, K Miyake, Nagisa Asakawa, M Yamamoto, Takashi Shimada · 2014 · Gene Therapy
5. AAV1/2-mediated BDNF gene therapy in a transgenic rat model of Huntington’s disease
   Bronwen J. Connor, Yunjuan Sun, Daniela von Hieber, S K Tang, K S Jones, Christof Maucksch · 2015 · Gene Therapy
6. Mechanisms of distribution of mouse β-galactosidase in the adult GM1-gangliosidosis brain
   Marike L. D. Broekman, Laryssa A. Tierney, Caroline Benn, P Chawla, J H, Miguel Sena‐Esteves · 2008 · Gene Therapy
7. Adeno-associated Virus Type 5 Reduces Learning Deficits and Restores Glutamate Receptor Subunit Levels in MPS VII Mice CNS
   Gumei Liu, Yong Hong Chen, Xiaohua He, Inês Martins, Jason Heth, John A. Chiorini, Beverly L. Davidson · 2007 · Molecular Therapy
8. Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy
   Heather L. Gray‐Edwards, Brandon L. Brunson, Merrilee Holland, Adrien‐Maxence Hespel, Allison M. Bradbury, Victoria J. McCurdy, Patricia M Beadlescomb, Ashley N. Randle, Nouha Salibi, Thomas S. Denney et al. · 2015 · Molecular Genetics and Metabolism
9. Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector
   Kohei Hironaka, Yoshiyuki Yamazaki, Yukihiko Hirai, Motoko Yamamoto, Noriko Miyake, Koichi Miyake, Takashi Okada, Akio Morita, Takashi Shimada · 2015 · Scientific Reports
10. Gene transfer to the CNS is efficacious in immune-primed mice harboring physiologically relevant titers of anti-AAV antibodies
    Treleaven, Christopher M, Tamsett, Thomas J, Bu, Jie, Fidler, Jonathan A, Sardi, S Pablo, Hurlbut, Gregory D, Woodworth, Lisa A, Cheng, Seng H, Passini, Marco A, Shihabuddin, Lamya S et al. · 2012 · Molecular Therapy
11. Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1
    Sea Young Yoon, Jessica H. Bagel, Patricia A. O’Donnell, Charles H. Vite, John H N Wolfe · 2015 · Molecular Therapy
12. Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders
    Kim M. Hemsley, John J. Hopwood · 2010 · Journal of Inherited Metabolic Disease
13. Broad Functional Correction of Molecular Impairments by Systemic Delivery of scAAVrh74-hSGSH Gene Delivery in MPS IIIA Mice
    Duncan, F Jason, Naughton, Bartholomew J, Zaraspe, Kimberly, Murrey, Darren A, Meadows, Aaron S, Clark, Kelly Reed, Newsom, David E, White, Peter, Fu, Haiyan, McCarty, Douglas M · 2015 · Molecular Therapy
14. Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity
    Federico Mingozzi, Hildegard Büning · 2015 · Frontiers in Immunology
15. NGF gene therapy activates neurons in the AD patient brain
    Hemi Malkki · 2015 · Nature Reviews Neurology
16. α-l-Iduronidase transport in neurites
    Fengtian Chen, Sandrine Vitry, Michaël Hocquemiller, Nathalie Desmaris, Jérôme Ausseil, Jean‐Michel Heard · 2006 · Molecular Genetics and Metabolism
17. Amyloidosis, Synucleinopathy, and Prion Encephalopathy in a Neuropathic Lysosomal Storage Disease: The CNS-Biomarker Potential of Peripheral Blood
    Bartholomew J. Naughton, F. Jason Duncan, Darren A. Murrey, Tierra Ware, Aaron S. Meadows, Douglas M. McCarty, Haiyan Fu · 2013 · PLoS ONE
18. Sustained striatal ciliary neurotrophic factor expression negatively affects behavior and gene expression in normal and R6/1 mice
    Eileen M. Denovan‐Wright, Marissa Attis, Edgardo Rodríguez-Lebrón, Ronald J. Mandel · 2008 · Journal of Neuroscience Research
19. AAV-Dominant Negative Tumor Necrosis Factor (DN-TNF) Gene Transfer to the Striatum Does Not Rescue Medium Spiny Neurons in the YAC128 Mouse Model of Huntington's Disease
    Laura Taylor Alto, Xi Chen, Kelly A. Ruhn, Isaac Treviño, Malú G. Tansey · 2014 · PLoS ONE