FDA opens path to approval for Huntington's gene therapy
The US Food and Drug Administration is allowing biotech company uniQure to reapply for the licensing of a gene therapy that could slow the progression of Huntington's disease...
A large biological molecule made up of a string of sub-units called amino acids. There are thousands of different proteins in the human body, each with a particular job. For example, haemoglobin carries oxygen around the blood.
The US Food and Drug Administration is allowing biotech company uniQure to reapply for the licensing of a gene therapy that could slow the progression of Huntington's disease...
by BioNews
Overproduction of a DNA repair protein called EXO1 can trigger DNA damage and increase chemotherapy sensitivity in human cancer cells, a new study has reported...
by BioNews
by Paula Macho Rueda and 1 others
A novel topical gene therapy for the treatment of a rare genetic skin disorder has been approved by the Medicines and Healthcare products Regulatory Agency...
by Dr Teja Rus
A transplant of genome-edited stem cells designed to protect healthy donor cells from cancer therapy has shown promise in patients with aggressive blood cancers...
by Yan Lau
A new type of CAR-T cell therapy has achieved complete remission in patients with blood cancers, using lower cell doses and without the need for chemotherapy preconditioning...
A new CRISPR-based approach has been designed to selectively eliminate human cells with disease-causing genes in vitro...
A long-term clinical trial of a gene therapy for a rare genetic form of deafness has demonstrated sustained hearing restoration, while a separate gene therapy for the same condition has received approval from the Food and Drug Administration...
Dr J Craig Venter, a biologist and entrepreneur known for his role in the race to sequence the human genome, has died at age 79...
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