Article
Chromosome Transplantation: Opportunities and Limitations
2024-03-15
Abstract excerpt
There are thousands of rare genetic diseases that could be treated with classical gene therapy strategies such as the addition of the defective gene via viral or non-viral delivery or by direct gene editing. However, several genetic defects are too complex for these approaches. These “genomic mutations” include aneuploidies, intra and inter chromosomal rearrangements, large deletions, or inversion and copy number...
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Identifiers and source
- Literature Corpus work
- 13707e11-84fd-5ecd-a298-62eaadd9d91a
- DOI
- 10.20944/preprints202403.0880.v1
