Genomic editing could repair the defective 21-hydroxylase gene and provide a cure for 21-hydroxylase deficiency, the most common CAH variant, eliminating the current need for constant patient intervention.
First-pass extracted concept
genomic editing for congenital adrenal hyperplasia
Candidate: concept label1 source documents5 linked claims
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Aliases
genetic therapies for CAH, genomic editing
Evidence Snippets
Supporting Sources
Linked Claims
CRISPR/Cas technology is presented as enabling genomic editing approaches for congenital adrenal hyperplasia.
Quoted textsource-backed
The advent of CRISPR/Cas technology has brought previously inconceivable treatment options to reality.
Delivery of genomic editing reagents to adrenocortical progenitor cells is a major challenge for CAH genomic editing strategies.
Quoted textsource-backed
There are a number of technologies within reach for CAH, however, delivery of the genomic editing reagents to the elusive adrenocortical progenitor cells remains challenging.
The complexity of CAH genetics has implications for the choice of genomic editing strategy.
Quoted textsource-backed
Here we discuss the complexity of CAH genetics, which has implications for choice of genomic editing strategy, and potential future strategies for the development of a cure of CAH.
Therapeutic options for congenital adrenal hyperplasia remain sub-optimal despite life-saving glucocorticoids.
Quoted textsource-backed
Despite life-saving glucocorticoids, therapeutic options for congenital adrenal hyperplasia (CAH) remain sub-optimal.
Genomic editing could repair the defective 21-hydroxylase gene and provide a cure for 21-hydroxylase deficiency.
Quoted textsource-backed
Genomic editing could repair the defective 21-hydroxylase gene and provide a cure for 21-hydroxylase deficiency, the most common CAH variant, eliminating the current need for constant patient intervention.