Dr. Tiziana Cremona

Cell specific gene editing for treatment of Alpha 1 antitrypsin deficiency

Short biography:

Dr.Cremona Tiziana Patrizia studied cellular and molecular biology at University of Rome Tor Vergata, she continued her education and received masters in Applied Biology at Biomedical Research at University of Rome La Sapienza. She also obtained a PhD in Biomedical Science at the University of Bern. In 2017 she got a fellowship at Harvard University for a period of two years.

She is currently a senior postdoctoral scientist working with Dr. Amiq Gazdhar at the Department of Pulmonary Medicine, Department for Biomedical Research University hospital and University of Bern.

Her Project “Cell specific gene editing for treatment of Alpha 1 antitrypsin deficiency” aims to provide a novel method of cell specific gene editing to prevent disease progression. These will be the first studies to definitively address the pertinent question rather than make correlations. We will identify the underlying pathways responsible for lung structure preservation.

Project Summary:

Alpha 1 antitrypsin (AAT) deficiency is a single gene mutation disease and leads to severe complications. 

With new advances in gene editing techniques, it will be possible to correct the mutation to restore the normal function of the gene. In the current proposal, I aim to test the novel and safe gene editing method to correct the mutation that leads to AATD. I will use the nonviral method of targeted delivery only to specific cells to achieve gene correction. This study will help us optimize the procedure and study any possible toxic effects of the gene-editing technique and lay grounds for precision medicine approach for treatment of Alpha 1 antitrypsin dependent emphysema.