Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia.
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Orphanet J Rare Dis. 2025 Jun 5;20(1):279. doi: 10.1186/s13023-025-03828-8.
Orphanet J Rare Dis. 2025.
PMID: 40474241
Free PMC article.
Review.