Developing gene knockdown-replacement therapies for spinocerebellar ataxia type 7
<p>For many dominant diseases, conventional treatment options are limited. This makes them attractive candidates for gene therapy, which may be directed to specifically silence a disease-causing allele. However, many mutations are not easily amenable to this technique, including nucleotide rep...
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| Format: | Thesis |
| Language: | English |
| Published: |
2013
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