Extensive preclinical research over the past 30 years has culminated in the recent regulatory
approval of several gene therapy products for hemophilia. Based on the efficacy and safety …

Potency is a key optimization parameter for hemophilia A gene therapy product candidates.
Optimization strategies include promoter engineering to increase transcription, codon …

Essentials• B cells are attractive targets for gene therapy and particularly interesting for
immunotherapy.• A baboon envelope pseudotyped lentiviral vector (BaEV‐LV) was tested …

Until recently, adeno-associated virus 9 (AAV9) was considered the AAV serotype most
effective in crossing the blood–brain barrier (BBB) and transducing cells of the central …

The delivery of nucleic acids relies on vectors that condense and encapsulate their cargo.
Especially nonviral gene delivery systems are of increasing interest. However, low …

The treatment landscape for hemophilia has been rapidly changing with introduction of
novel therapies. Gene therapy for hemophilia is a promising therapeutic option for sustained …

The robustness and safety of liver-directed gene therapy can be substantially improved by
enhancing expression of the therapeutic transgene in the liver. To achieve this, we …

Hemophilia A (HA), a common bleeding disorder caused by a deficiency of coagulation
factor VIII (FVIII), has long been considered an attractive target for gene therapy studies …

The development of robust nonviral vectors could facilitate clinical gene therapy applications
and may overcome some of the immune complications of viral vectors. Nevertheless, most …

Over 50 years after its initial description, adeno-associated virus (AAV) remains the most
exciting but also most elusive study object in basic or applied virology. On the one hand, its …