Author Interviews, Leukemia, Stem Cells, Technology / 11.02.2021

MedicalResearch.com Interview with: Eirini Papapetrou, MD, PhD Associate Professor Department of Oncological Sciences Icahn School of Medicine at Mount Sinai New York, NY 10029 MedicalResearch.com: What is the background for this study? Would you tell us a little about acute myeloid leukemia? Response: Acute myeloid leukemia is a form of cancer of the blood. It is typically very aggressive and lethal without treatment. The main treatment is high-dose chemotherapy and it has not changed very much in decades. Some more recent "targeted" therapies that are less toxic help somewhat but still do not result in cures. We believe a reason for this might be that both chemotherapy and newer "targeted" therapies target the cells at the later stages of the disease and spare the earlier ones, which can then give rise to disease resistance and relapse.  (more…)
Author Interviews, Diabetes, Genetic Research, Personalized Medicine / 27.04.2020

MedicalResearch.com Interview with: Fumihiko Urano, MD, PhD Samuel E. Schechter Professor of Medicine Division of Endocrinology, Metabolism, and Lipid Research Washington University School of Medicine MedicalResearch.com: What is the background for this study? Response: Wolfram syndrome is a rare monogenic disease characterized by insulin-dependent diabetes, retinal degeneration, and neurodegeneration. Using gene editing by CRISPR-Cas9, in combination with patient-derived induced pluripotent stem cells (iPSCs), we were able to make normal insulin-producing pancreatic beta cells by correcting Wolfram Syndrome gene mutation. We could cure diabetes in cells and mice. Because we can create any types of tissues from iPSCs, our next step would be to replicate this success for other medical problems, including retinal regeneration and neurodegeneration. (more…)
Author Interviews, Cancer Research, Genetic Research / 08.04.2019

MedicalResearch.com Interview with: Eunhee Yi, Ph.D. Postdoctoral Associate The Jackson Laboratory MedicalResearch.com: What is the background for this study? What are the main findings?  Response: Recurrence after therapy for glioblastoma (GBM) is unavoidable. There are substantial differences among the cells of GBM tumors in the abundance and types of genetic materials. This heterogeneity is a major driver of therapy failure and disease progression. We previously reported that extrachromosomal DNA (ecDNA) elements, which reside outside the linear genome and represent a mechanism to amplify and activate oncogenes, is a potential cause of the increasing genetic diversity in GBM. Our current study is focused on the development of a novel cytogenetic tool to visualize ecDNA to visualize the behavior of these elements in live cells. We have leveraged the unique properties of ecDNA to develop a CRISPR-based “ecDNA tracing toolbox (EDTB)”.  (more…)
Author Interviews, Ophthalmology / 23.04.2017

MedicalResearch.com Interview with: Kang Zhang, M.D., Ph.D. Professor of Ophthalmology Chief, Ophthalmic Genetics Founding Director, Institute for Genomic Medicine Co-Director, Biomaterials and Tissue Engineering, Institute for Engineering in Medicine Board Certification in Ophthalmology Fellowship in Vitreoretinal Disease and Surgery Guangzhou Women and Children's Medical Center Guangzhou Medical University Guangzhou China MedicalResearch.com: What is the background for this study? What are the main findings? Response: Retinitis pigmentosa is a common blinding condition characterized by mutations in rod photoreceptor specific genes, night blindness and tunnel visual with eventual loss of day vision. Since it can be caused by numerous different mutations in many genes therefore it has been difficult to provide treatment benefits to a majority of patients. Traditional gene therapy has been in a piece-meal fashion, meaning to create a therapy for a particular gene or mutation. In this paper, we describe a universal gene therapy approach using the latest gene editing technology CRISPR/CAS9 to reprogram rod photoreceptors to cone photoreceptors with reversal of RP and restoration of vision. (more…)