Dr. Louise Rodino-Klapac of Sarepta Therapeutics and Livija Medne of the Children’s Hospital of Philadelphia discuss limb-girdle muscular dystrophy (LGMD).
All tagged gene therapy
#94 Legend of Sumeria: A Genetics Graphic Novel
Dr. Biju Parekkadan, Co-Creator and Scientific Director of Legend of Sumeria, discusses the science, bioethics, and art behind this hard science graphic novel.
#88 Ira Pastor on Regeneration
The CEO of Bioquark shares on the advancements in regeneration and repair of human organs and tissues.
#83 Alex Ille on Alzheimer's Disease
Genome Cure Organization’s Director shares the genetics of Alzheimer’s Disease and recent research.
#44 CRISPR
CRISPR is a genetic editing technology that will change the future of genetics. In the past 3 years it has been used in labs throughout the world. It has the potential to fix point mutations and larger mutations in our genome. Diseases caused by point mutations include Cystic Fibrosis, Sickle Cell Anemia, and Tay-Sachs disease. More complex conditions such as cancer, HIV or autism could be cured if the RNA gene editing is developed further.
#7 Gene Therapy
Is gene therapy ethical and worth pursuing as a treatment?