Guest Editors
Katarzyna Dorota Raczynska
Adam Mickiewicz University
Prof. Katarzyna Dorota Raczynska, is a laboratory head at the Adam Mickiewicz University. Our laboratory research is focused on RNA processing mediated by U7 snRNP. Her lab studies sdRNAs and the novel functions of U7 snRNA; studying the interaction between hnRNP UL1, FUS, and U7 snRNP in human cells; to understand the effect of ALS-linked FUS mutations on U7 snRNP activity; to identify the small RNA's derived from mature snoRNAs and to elucidate the molecular mechanisms underlying their synthesis mediated by the FUS protein.
Ankur Gadgil
Adam Mickiewicz University, Poland
Ankur Gadgil is currently a PhD student at the Adam Mickiewicz University in Poland. His PhD topic is focused on studying the effect of ALS-FUS mutations on U7 snRNP activity. He was recently awarded the EMBO short-term fellowship for 3 months wherein he studied the involvement of FUS and U7 snRNP in prostate cancer. His scientific background includes a masters in virology and a one-year experience as a project fellow to study the role of keratins, vimentins, and associated proteins in the prognosis of human oral cancer. Moreover, he has worked in the industry for one year and developed an ELISA-based detection kit for the asymptomatic detection of bacterial blight on the pomegranate plant. His research exchange at King’s College London and the University of Eastern Finland has helped him to gain experience in international communication skills, working in diverse teams, and promptly adapting to different environments.
Collection Overview
The concept of gene therapy is based on the idea of treating a genetic problem at it source. In this concept, nucleic material is delivered into the cell to treat a disease. Genetic diseases and acquired genetic disorders can be treated using gene therapy. Treatment using gene therapy works in many ways. For instance, gene therapy can replace a faulty gene, silence the disease-causing gene, or introduce a new gene to repair or restore normal function of the faulty gene. As with any other upcoming technology, gene therapy has its own limitations such as the delivery of the construct, immunoreactivity, and the timing of the start of therapy. With the advancement of technologies such as CRISPR/Cas9, engineering the cells in vivo and transplanting the corrected cells into the patient is a promising approach. In this collection we will look at the methods and technologies involving cellular engineering, including somatic gene therapy, germ line gene therapy, in vivo or in vitro cell engineering, viral or non-viral vector delivery, stem cell engineering and autologous transplantation, and the use of CRISPR/Cas9 in gene therapy.
Articles
Assessment of Chimeric Antigen Receptor T Cell-Associated Toxicities Using an Acute Lymphoblastic Leukemia Patient-Derived Xenograft Mouse Model
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Cited by 4
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2023
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Claudia Manriquez Roman1,2,3,4,5, R. Leo Sakemura1,2, Brooke L. Kimball1,2, Fang Jin6, Roman H. Khadka6, Mohamad M. Adada1,2, Elizabeth L. Siegler1,2, Aaron J. Johnson6, Saad S. Kenderian1,2,6
1T Cell Engineering Laboratory, Mayo Clinic, Rochester, 2Division of Hematology, Mayo Clinic, Rochester, 3Mayo Clinic Graduate School of Biomedical Sciences, Mayo Clinic, Rochester, 4Department of Molecular Medicine, Mayo Clinic, Rochester, 5Regenerative Sciences PhD Program, Mayo Clinic, Rochester, 6Department of Immunology, Mayo Clinic, Rochester