The reputation of stem cell research is being undermined by dodgy clinics offering unrealistic therapies for unproven diseases.
Spark Therapeutic’s Luxturna, a gene therapy for a rare, genetic form of blindness, will be reviewed by a U.S. Food and Drug Administration panel this week.
For the first time, scientists have documented a direct link between deletions in two genes, linked to multiple brain and body disorders.
Cancer researchers have discovered a new class of fusion genes with properties that affect and may drive the development of cancer.
Cambridge gene therapy firm Dimension Therapeutics has accepted a $151 million deal that will allow them to maintain a local presence.
The first gene therapy for cancer, approved by the FDA in August, will transform the treatment process, but it will bring both hope and challenges.
George Church has co-founded a new startup that aims to develop “designer proteins” targeting a range of disorders.
UK scientists have edited the DNA of human embryos for the first time, helping them further understand infertility and miscarriage.
Disorder in 1 gene triggers a chain reaction that disrupts the construction and stability of the brain’s developing connections
Interested in how film making can work as rare disease advocacy? Come and meet Daniel DeFabio at the Festival of Genomics Boston on 3-4 October 2017.
Researchers healed bone fractures by attracting stem cells to the area and injecting a mix of microbubbles and DNA encoding a bone protein at the break.