The aim of targeted gene-based cancer therapies could often be skewed from the start. A widespread concept about how cells produce proteins proved incorrect 62 percent of the time in a new study in ovarian cancer cells on the relationship between RNA and protein levels.
Scientists have edited the pig genome to deactivate a family of retroviruses. The results hold important implications for transplant medicine in humans.
Clinicians today have a huge arsenal of drugs at their disposal for treating cancers. But many chemotherapeutic agents pose stubborn challenges: they cause serious side effects, some cancers develop resistance, and many chemotherapies demonstrate low bio-availability. A potential solution lies in the synergistic combination of a chemotherapeutic drug with engineered genetic material. New hybrid materials developed at NYU Tandon combine a lipid 'container' for transfection and a protein capsule to deliver a chemical one-two punch.
A new Hastings Center special report examines efforts to revive extinct species.
An international group of 11 organizations with genetics expertise has issued a policy statement on germline genome editing in humans, which recommends against genome editing that culminates in human pregnancy; supports publicly funded, in vitro research into its potential clinical applications; and outlines scientific and societal steps necessary before implementation of such clinical applications is considered.
A research team has overcome challenges that have limited gene therapy. They demonstrate how their novel approach with skin transplantation could enable a wide range of gene-based therapies to treat human diseases. The researchers provide 'proof-of-concept,' treating mice with two common related human ailments: type-2 diabetes and obesity.
Published in Nature, CRISPR-Cas9 genetic scissors open up new pathways to treat and prevent genetic diseases.
Scientists achieve first safe repair of single-gene mutation in human embryos.
Researchers at Purdue University have discovered a mechanism for delivering tumor-suppressing microRNAs that eliminates the need for toxic delivery vehicles.
Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers have developed a new gene transfer approach that uses an adeno-associated virus vector to deliver a modified dystrophin gene to muscle, restoring muscle strength in a mouse model that closely mimics the severe defects seen in patients. The study appears July 27 in Molecular Therapy - Methods & Clinical Development.