The New England Journal of Medicine

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

The goal of gene therapy for patients with hemophilia A is to safely impart long-term stable factor VIII expression that predictably ameliorates bleeding with the use of the lowest possible vector ...
TMCnet

Flipping the script: Scripta Therapeutics raises $12M to find disease-modifying drugs that alter transcription factor activity

Scripta's unique approach is based on the concept that transcription factors, the master controllers of gene expression, are core drivers of disease and act as detailed and dynamic maps of the ...
FierceBiotech

Expression Therapeutics LLC Selects ABL, Inc. to Support Cell Line Development for Hemophilia A Factor VIII Infusion Therapy

TUCKER, Ga. & ROCKVILLE, Md.--(BUSINESS WIRE)--Expression Therapeutics LLC, a biotechnology company developing high expression factor VIII technologies, together with ABL, Inc., a biomedical contract ...
Nature

Expression of CD33 is a predictive factor for effect of gemtuzumab ozogamicin at different doses in adult acute myeloid leukaemia

Results from the Children’s Oncology Group (COG) AML trials showed that benefit from GO at a single dose of 3 mg/m 2 at first induction and then intensification 9 was restricted to paediatric patients ...

Transcription factor drives chemotherapy resistance in ovarian cancer

Northwestern Medicine scientists have discovered how a specific transcription factor promotes genetic reprogramming and chemotherapy resistance in ovarian cancer cells, findings that may inform new ...