Dual SORT LNPs for multi-organ base editing.

Alpha-1 antitrypsin (A1AT) deficiency (AATD) is caused by a mutation in the SERPINA1 gene (PiZ allele), where misfolded A1AT liver accumulation leads to liver damage, and A1AT deficiency in the lungs results in emphysema due to unregulated neutrophil elastase activity. Base editing offers a potential cure for A1AT; however, effective treatment is hindered by the absence of dual-target delivery systems that can target key tissues. We developed Dual Selective ORgan-Targeting lipid nanoparticles (SORT LNPs) to deliver base editors to the liver and lungs.

Factors affecting patient presentation at a national dermatology referral clinic in Kampala, Uganda.

Background: This study sought to gain a better understanding of the patient population in Kampala and was further designed to elucidate barriers that may delay individuals from receiving proper dermatologic care.

Methods: The study took place at the dermatovenereology clinic of a tertiary care hospital in Kampala. New adult patients were surveyed in July and August of 2013.