Litcius/Paper detail

Hemophilia as a blueprint for gene therapy

Margaret V. Ragni

2021Science17 citationsDOI

Abstract

Advances in hemophilia gene therapy could enable progress in other inherited disorders.

Topics & Concepts

Genetic enhancementBlueprintComputational biologyGeneMedicineBioinformaticsGeneticsBiologyEngineeringMechanical engineeringHemophilia Treatment and Research