Altering Tropism of rAAV by Directed Evolution
Fig. 1 Library enrichment for target-specific variants. The DNA encoding the initial capsid library (top left) is cloned into the vector and enters the cycle through which target-specific variants are…
AAV-Mediated Gene Transfer to Dorsal Root Ganglion
Fig. 1 Intraganglionic injection. Paravertebral surgical exposure for intraganglionic injection, illustrating the operative field (left panel), reference diagram (middle panel), and cleaned vertebral bones (right panel). (a) Initial dissection of…
Delivering Transgenic DNA Exceeding the Carrying Capacity of AAV Vectors
Fig. 1 Overlapping AAV genome design and transduction. (a) The transgenic cassette, containing the depicted elements, is split into two separate vectors such that there is a homologous sequence overlap…
AAV2-Neurturin for Parkinson’s Disease: What Lessons Have We Learned?
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_32 32. AAV2-Neurturin for Parkinson’s Disease: What Lessons Have We Learned? Jeffrey H. Kordower1 (1) Department of Neurological Sciences, Rush…
Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_30 30. Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders Dominic J. Gessler1 and Guangping Gao1 (1) University of…
Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis
Route of administration Advantages Disadvantages Intramuscular (IM) Noninvasive Requires retrograde transport of the vector; impractical to treat all skeletal muscles Intraparenchymal (IP) Delivery of therapeutic directly to location of interest…
Gene Therapy-Based Modeling of Neurodegenerative Disorders: Huntington’s Disease
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_27 27. Gene Therapy-Based Modeling of Neurodegenerative Disorders: Huntington’s Disease Deborah Young1 (1) Department of Pharmacology & Clinical Pharmacology…
Systemic Gene Therapy for Targeting the CNS
Fig. 1 Representative images of mouse brain following systemic injection of AAV9 CB GFP. GFP immunofluorescence shows extensive GFP expression in neurons throughout multiple structures including the cortex (A), hippocampus…
Tissue-Specific Promoters in the CNS
Fig. 1 Transcriptional targeting of AAV vectors in the rat striatum. Exclusively neuronal transgene expression is shown after AAV-5-hSYN-EGFP transduction (left panel; shown is an overlay of green = EGFP fluorescence with…
Non-Viral, Lipid-Mediated DNA and mRNA Gene Therapy of the Central Nervous System (CNS): Chemical-Based Transfection
© Springer Science+Business Media New York 2016Fredric P. Manfredsson (ed.)Gene Therapy for Neurological DisordersMethods in Molecular Biology138210.1007/978-1-4939-3271-9_23 23. Non-Viral, Lipid-Mediated DNA and mRNA Gene Therapy of the Central Nervous System (CNS): Chemical-Based Transfection James G. Hecker1 …
