Principles of Gene Therapy Applicability
Terms of Applicability
Type of Inheritance
Mendelian diseases, determined by the action of a single gene, are better candidates than multifactorial diseases, which rely on the joint action of several genes and are influenced by environmental factors.
Pattern of Inheritance
Recessive diseases are better candidates for gene therapy treatment than dominant ones.
Nature of the Mutation
- Recessive Mutations: When the affected gene produces an inadequate protein, the introduction of a normal copy of the gene is sufficient to produce a functional protein and correct the condition.
- Dominant Mutations: Replacing one gene is insufficient because the mutant gene continues to be read and translated. These require more complex strategies, such as blocking the specific