Share this story

UMass Chan gene therapy experts to present new discoveries at inKNOWvation panel discussion

Scientists at UMass Medical School will share their latest research at an inKNOWvation panel discussion, “The Return of Gene Therapy,” on Wednesday, May 11, at the Massachusetts Biotechnology Council in Cambridge.

“When gene therapy was initially talked about, it was a simple concept: replace a bad gene with a good one. The field has become much more complicated as it has advanced,” said Guangping Gao, PhD, the Penelope Booth Rockwell Professor in Biomedical Research, professor of microbiology & physiological systems and director of the Horae Gene Therapy Center & Vector Core at UMMS.

Dr. Gao will open the event with a short presentation on the current state of gene therapy. Gao said that in recent years, the National Institutes of Health has increased funding for gene therapy-related research and clinical trials and that venture capital and pharmaceutical company investment in gene therapy research has doubled year over year since 2013. There has been rapid and solid progress in research to understand both disease mechanisms and the way to interfere with the course of disease, Gao said. There has also been greater public awareness and understanding of rare diseases, such as amyotrophic lateral sclerosis (ALS), Canavan, Tay-Sachs and Alpha-1 antitrypsin deficiency.

The field of gene therapy has evolved from gene replacement to gene silencing to gene addition and is now entering a fourth stage, one that enables gene editing. Along the way, basic and translational science researchers at UMMS have significantly contributed to this scientific growth, including discoveries by:

      • Nobel Laureate Craig Mello, PhD, Howard Hughes Medical Institute Investigator, the Blais University Chair in Molecular Medicine and distinguished professor of molecular medicine and cell & developmental biology, who co-discovered RNA interference with Andrew Z. Fire, PhD;
      • Phillip D. Zamore, PhD, Howard Hughes Medical Institute Investigator, the Gretchen Stone Cook Chair in Biomedical Sciences, professor of biochemistry & molecular pharmacology, and founding chair of the RNA Therapeutics Institute, who played a major role in translating RNAi to experimental medicine;
      • Robert H. Brown Jr., DPhil, MD, the Leo P. and Theresa M. LaChance Chair in Medical Research, chair and professor of neurology, who is at the forefront of ALS research and associated gene therapy, and, in 1993, led a team of researchers that discovered the first gene linked to the inherited form of ALS;
      • Michael Green, MD, PhD, a Howard Hughes Medical Institute Investigator; the Lambi and Sarah Adams Chair in Genetic Research; director of the UMass Medical School Cancer Center; and chair and professor of molecular, cell & cancer biology, who uses transcription-based approaches and functional screens to identify new genes and regulatory pathways involved in cancer, and translation of those discoveries into novel cancer therapeutics;
      • Miguel S. Sena-Esteves, PhD, assistant professor of neurology, a leading force behind the creation of the Tay-Sachs Gene Therapy Consortium; and
      • Chris Mueller, PhD, associate professor of pediatrics, whose research with rAAV shows persistent gene expression over long periods of time, demonstrating the efficacy of the approach.

Drs. Brown, Green, Sena-Esteves, Mueller and Gao will participate in a panel discussion focused on gene therapy research moderated by Terence R. Flotte, MD, the Celia and Isaac Haidak Professor of Medical Education, executive deputy chancellor, provost and dean of the School of Medicine.

Dean Flotte was the principal investigator for the first use of recombinant AAV in humans, using the vector in cystic fibrosis trials in 1995, before shifting his emphasis to alpha-1 antitrypsin deficiency, a genetic cause of emphysema. Flotte’s studies established important safety data and contributed seminal observations regarding the molecular mechanisms of rAAV persistence and immune response in humans.

InKNOWvation is a scientific networking series that highlights the latest discoveries at UMMS in an interactive setting to cultivate engagement, discussion and collaboration among top scientists and the region’s thought leaders in science, medicine, education and business.

The event takes place on May 11 from 5 to 8 p.m. at MassBio, located at 300 Technology Square, 8th floor, in Cambridge. For additional information and to register, visit the inKNOWvation event web page.