High-efficiency base editing in the retina in primates and human tissues.

Stargardt disease is a currently untreatable, inherited neurodegenerative disease that leads to macular degeneration and blindness due to loss-of-function mutations in the ABCA4 gene. We have designed a dual adeno-associated viral vector encoding a split-intein adenine base editor to correct the most common mutation in ABCA4 (c.5882G>A, p.

Significance of cycle 1 bone marrow biopsy in predicting outcomes and toxicities of venetoclax-based therapy for myeloid malignancies.

For older unfit patients receiving venetoclax-based induction, data on the significance of interim bone marrow biopies (BMBx) findings on clinical outcomes is lacking. We retrospectively evaluated interim BMBx results performed on Cycle 1 days 21-28 of venetoclax-based therapy in 69 adults with myeloid malignancies to determine whether blast clearance was associated with overall survival (OS) and overall response rate (ORR). Median age was 75 years (range 69-78).

What impact does widening participation to medicine have on the medical workforce in the UK: a scoping review.

Purpose: Widening participation in medicine refers to the recruitment policy of encouraging those who are traditionally under-represented in medical school. Whilst research in the UK has investigated the processes around improving participation through recruitment and selection to medical schools, there is less focus around the period after medical school and how students from widening participation backgrounds fare in the workforce.

Methods: This study employed scoping review methodology to collate, map and summarise research in the field.