gene therapy, culture, cell & molecular biology, ecology
Families of Children with Rare Diseases Fuel Gene Therapy Research
Families of Children with Rare Diseases Fuel Gene Therapy Research
The Scientist Staff | May 1, 2018
Crowdfunding can power investigations into diseases that would otherwise receive little attention.
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
A Devastating Diagnosis, a Viral Video, and a Clinical Trial
Diana Kwon | May 1, 2018
When the O’Neills learned that their daughter had Sanfilippo syndrome, a devastating rare disease, they created a GoFundMe campaign that raised $2 million in less than a year.
The Philadelphia Family Helping to Drive Research on MLD
The Philadelphia Family Helping to Drive Research on MLD
Catherine Offord | May 1, 2018
Though Calliope Joy’s disease is too far progressed to be treatable, her parents have helped other children with metachromatic leukodystrophy get access to an experimental therapy.
Slow March Toward a Canavan Cure
Slow March Toward a Canavan Cure
Ashley Yeager | May 1, 2018
Two decades after a successful crowdfunding campaign, some clinical trial patients have seen improvements—but there’s still no approved treatment for the disease.
Infographic: Piecing the Cholesterol Puzzle
Infographic: Piecing the Cholesterol Puzzle
Diana Kwon | May 1, 2018
How a rare disease led to an understanding of the basics of cholesterol regulation.
Trailblazing Endocrinologist Neena Schwartz Dies
Trailblazing Endocrinologist Neena Schwartz Dies
Kerry Grens | Apr 27, 2018
The reproductive biologist uncovered hormones important for fertility cycles.
Worms’ Magnetic Sense Questioned
Worms’ Magnetic Sense Questioned
Abby Olena | Apr 25, 2018
Unsuccessful attempts to reproduce the results of a 2015 study reporting that C. elegans orient themselves by Earth’s magnetic field spark debate among researchers.
Salk Institute Suspends Cancer Scientist Inder Verma
Salk Institute Suspends Cancer Scientist Inder Verma
Ashley Yeager | Apr 23, 2018
The prominent researcher has been put on administrative leave pending an investigation into unspecified allegations.
Image of the Day: Nanobot Schematic
Image of the Day: Nanobot Schematic
The Scientist Staff | Apr 13, 2018
A magnetically controlled device could have applications in studies of cell biology and biophysics.
World’s Largest Cell and Gene Therapy Plant Opens
World’s Largest Cell and Gene Therapy Plant Opens
Catherine Offord | Apr 11, 2018
Lonza will employ more than 200 full-time staff to work at the Texas-based facility, the company says.