Explores gene regulation through transcription mechanisms and chromatin accessibility, emphasizing the importance of transcription factors and gene regulatory networks.
Explores the production of viral vectors for gene therapy applications, emphasizing AAV vectors and the process of large-scale production and purification.
Explores gene regulation mechanisms, including chromatin accessibility, DNA looping, and transcription factors' role in controlling cellular functions.
Explores preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.
