Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients

By sjmartinez • November 14, 2024

Emami MR, Brimble MA, Espinoza A, Owens J, Whiteley LO, Casinghino S, Lanz TA, Farahat PK, Pellegrini M, Young CS, Thomas PG, McNally EM, Villalta SA, Schattgen SA, Spencer MJ. Single cell and TCR analysis of immune cells from AAV gene therapy-dosed Duchenne muscular dystrophy patients. Mol Ther Methods Clin Dev. 2024 Sep 30;32(4):101349. doi: 10.1016/j.omtm.2024.101349. PMID: 39524974; PMCID: PMC11546459.

Tags: AAV, adeno-associated virus, Dermatology, Duchenne, Gene therapy, immune response, muscular dystrophy, neuromuscular disorders, Publications, single-cell sequencing, T cell receptor sequencing

Treatment Patterns in Pediatric Hidradenitis Suppurativa: Time to Treatment Initiation, Initial Treatment, and Time to Switching

Aflatooni S, Ure A, Smart K, Cortes JA, Palma G, Johnsen N, Nong Y, Armstrong AW. Treatment Patterns in Pediatric Hidradenitis Suppurativa: Time to Treatment[...]

Photosensitive symmetric papules of the nasal alae

Peng DS, Dong E, Seivright J, Gates G, Nguyen MO. Photosensitive symmetric papules of the nasal alae. JAAD Case Rep. 2026 Jan 21;70:253-255. doi: 10.1016/j.jdcr.2026.01.017.[...]

International development of Investigator Global Assessment scores for pemphigus

Murrell DF, Boucher D, Daneshpazhooh M, Mahmoudi H, Kianfar N, Dasdar S, Yale M, Wilson A, Bilgic A, Uzun S, Chandran NS, Patsatsi A, Drenovska[...]