| Murlidharan et al. |
Describes AAV-vector biology, their cellular entry mechanisms and axonal transport profiles of well-characterized AAV serotypes. Discusses the implications of AAV-vector applications (e.g., direct application, intravenous injections, etc.). Considers the safety aspects of AAV-mediated applications to the CNS. |
Review |
| Parr-Brownlie et al. |
Describes lentiviral vector biology, including modified envelope glycoproteins and the expression of transgenes under the regulation of cell-selective and inducible promoters. Deliberates on the benefit of lentiviral-vectors combined with other techniques such as anatomical tract-tracing, immunohistochemistry, confocal and electron microscopy. Proposes limitations and future perspectives including ways that lentiviral-vectors can contribute to the gene therapy clinical trials. |
Review |
| Tan et al. |
Explores the challenges facing non-viral nucleic acid delivery to the CNS and provides strategies to overcome them. Discusses the advantages and disadvantages of different administration routes of nucleic acid delivery. Considers how retrograde axonal transport can be used to deliver non-viral nucleic acids. |
Review |
| Wagner et al. |
Describes the epidemiology, molecular pathology and mouse models related to spinocerebellar ataxia-1 (SCA-1). Discusses the literature related to stem cell, gene and alternative therapies used to treat SCA-1. Identifies the various challenges for gene, stem cell and alternative therapies for SCA-1. |
Review |
| Tosolini and Sleigh |
Describes the epidemiology, genetics, classifications and mechanisms causing SMA and ALS/MND and deliberates on potential commonalities. Provides an update on clinical gene therapies for both SMA and ALS/MND. Identifies four key areas that ALS/MND gene therapies can learn from the recent success in the SMA gene therapies including therapeutic targeting, combinational treatment, considering the dose and drug concentration as well as optimizing the therapeutic timing. |
Review |
| Craig and Housley |
Provides a summary of the viral-mediated gene therapy research used to treat stroke. Highlights the key areas that gene therapy needs to address to ameliorate stroke including protein synthesis, delivery site and viral-vectors. Identifies therapeutic protein candidates for stroke treatment. |
Mini-review |
| Stoica and Sena-Esteves |
Summarizes the literature on AAV-mediated gene therapy studies that reduce SOD1 toxicity to treat SOD1-related ALS/MND. Discusses the current hurdles to be addressed to advance the development of clinical gene therapies such as non-cell autonomous toxicity, cellular and anatomical targeting and the delivery methods. Identifies RNA interference as a successful therapeutic target to ameliorate disease. |
Mini-review |
| Yang et al. |
Summarizes the development and application of the CRISPR/CAS9 toolkit. Describes the use of CRISPR/Cas9 to generate animal models of neurodegenerative diseases. Discusses how CRISPR/Cas9 can be applied to treat animal models of Parkinson's and Huntington's Disease. |
Mini-review |
| Hoyng et al. |
Summarizes the research on gene therapy in animal models of peripheral nerve repair and identify key future directions. Provides a perspective on the path for clinical translation for PNS-gene therapy for traumatic nerve injuries. Addresses efficacy and safety concerns for human applications and identify the ideal patient population for a proof-of-concept clinical study. |
Hypothesis and theory |
