Publicaties
Molecular Tools for Editing Globin Genes Vrije Universiteit Brussel
The latest, novel developments in the field of gene editing have helped in gaining traction to correct diseases of genetic origin. New DNA analysis techniques and gene editing can help in ameliorating hemoglobinopathies. This chapter will cover methods of genome editing (ZFNs, TALENs, CRISPR/Cas9) with various aspects of gene delivery using viral (adenoviral, adeno-associated viral and lentiviral) vectors and nanovectors. Additionally, ...
In utero CRISPR-mediated therapeutic editing of metabolic genes Vrije Universiteit Brussel
In utero gene editing has the potential to prenatally treat genetic diseases that result in significant morbidity and mortality before or shortly after birth. We assessed the viral vector-mediated delivery of CRISPR-Cas9 or base editor 3 in utero, seeking therapeutic modification of Pcsk9 or Hpd in wild-type mice or the murine model of hereditary tyrosinemia type 1, respectively. We observed long-term postnatal persistence of edited cells in ...