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 preclinical and clinical trials in SMA gene therapy, including efficiency in patients, trial results, adverse effects, cellular targeting, and future perspectives.
Explores transcription factors in reprogramming somatic cells to induced pluripotent stem cells and the correlation between Hox gene expression and morphological boundaries.