Concepedia

Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I

Christian Hinderer, Peter Bell, Brittney L. Gurda, Qiang Wang, Jean‐Pierre Louboutin, Yanqing Zhu, Jessica Bagel, Patricia O’Donnell, Tracey Sikora, Therese Ruane,

Molecular Therapy · 2014 · 109 citations · 27 references

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References

27

Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

Michael G. Kaplitt, Andrew Feigin, Chengke Tang et al. · The Lancet · 2007 · 1.1K citations

, , +17

Mannose 6-Phosphate Receptors and Lysosomal Enzyme Targeting

Nancy Dahms, Peter Lobel, Stuart Kornfeld · Journal of Biological Chemistry · 1989 · 478 citations · Full text

, , +14

Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates

Steven J. Gray, Sahana Nagabhushan Kalburgi, Thomas J. McCown et al. · Gene Therapy · 2013 · 372 citations

, , +14

Long-Term Evaluation of a Phase 1 Study of AADC Gene Therapy for Parkinson's Disease

Gabriele Mittermeyer, Chadwick W. Christine, Kathryn H. Rosenbluth et al. · Human Gene Therapy · 2012 · 299 citations · Full text

, , +17

Immune responses to AAV in a phase I study for Canavan disease

Scott McPhee, C. G. Janson, Chuang Li et al. · The Journal of Gene Medicine · 2006 · 236 citations

, , +15