Genome Editing using AAV Vectors: A Synergistic Approach for Rare Disease Treatment

Over the past ten years, there has been a notable rise in genome editing tools boasting diverse effectiveness and promising prospects for treating conditions both within and outside the body. When paired with a harmless viral vector such as AAV, this approach becomes particularly potent in addressing rare and ultra-rare disorders, which often pose numerous challenges due to multiple factors. A thorough examination of the various viral vectors presently employed will furnish the scientific community with crucial insights for informed decision-making, aiding in the selection of customized genome editing solutions to maximize the benefits for patient populations.

This Research Topic aims to investigate three crucial facets of genome editing using AAV, as outlined below:

1. Effectiveness: AAV vectors are recognized for their adept delivery of genomic material, whether in single or dual vector configurations. Enhancing and sustaining the efficiency of genome editing via AAV vectors is paramount, given the multitude of rare disorders requiring therapeutic intervention.

2. Suitability: AAV-mediated genome editing holds promise in tailoring treatments to individual disorders based on the specific organs affected. This aspect remains underexplored and merits further exploration to advance personalized medicine solutions.

3. Management of Adverse and Severe Adverse Events (AE and SAE): While AAV-based genome editing presents significant benefits, the potential for AE/SAE due to factors such as loss of efficacy or the choice of editing tools must be carefully considered against the urgent need for effective therapies.

This Research Topic welcomes original research articles, reviews, vector production (method development and analytical assays), and case studies that cover the following (but are not limited to) topics:

Specific Themes should involve (but are not limited to) the following:

1. AAV-mediated genome editing for blood disorders: Advantages and limitations
2. AAV-mediated genome editing: Vector design, tissue targeting, & assays for detection
3. AAV-mediated genome editing: Establishing animal models and pre-clinical tests
4. AAV-mediated genome editing: Current vector production methods
5. AAV-mediated genome editing: Ethical aspects and involvement of patient groups

Keywords: Genome Editing, Adeno-Associated Virus, CRISPR Cas, Zinc Finger Nucleases, TALEN

Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.