Off-target edits, where CRISPR cuts DNA at unintended locations, pose a significant risk of disrupting vital genes and potentially leading to conditions like cancer. Non-viral methods, such as lipid nanoparticles (LNAs) and polymer-based carriers, are also being developed to reduce immunogenicity and improve cargo capacity.
In Vivo CRISPR Clinical Progress Review: Current Advances and Safety Challenges
Other targets include sickle cell disease and certain metabolic liver conditions, where the goal is to correct a single-gene defect in hepatocytes to restore normal physiological function. Viral vectors, particularly adeno-associated viruses (AAVs), are currently the leading delivery method due to their ability to efficiently enter cells and provide long-term expression.
A specific guide RNA (gRNA) is designed to locate a precise DNA sequence, while the Cas9 enzyme acts as molecular scissors to create a targeted cut. Immune reactions to the Cas9 protein or delivery vector can diminish efficacy or cause adverse effects.
In Vivo CRISPR Clinical Progress Review: Navigating Delivery and Safety Hurdles
Advanced techniques allow for epigenetic modulation, turning genes on or off without altering the underlying DNA sequence. The primary challenge lies in delivering this molecular machinery safely and efficiently to the intended cell types without triggering an immune response or causing off-target effects.
More About In vivo crispr
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