Lentiviral vectors are among the most effective viral vectors for vaccination. In clear contrast
to the reference adenoviral vectors, lentiviral vectors have a high potential for transducing …

Sickle cell disease (SCD) is a monogenic disorder characterized by recurrent episodes of
severe bone pain, multi-organ failure, and early mortality. Although medical progress over …

Diamond-Blackfan anemia (DBA) is a genetic blood disease caused by heterozygous loss-
of-function mutations in ribosomal protein (RP) genes, most commonly RPS19. The …

Introduction Ex vivo gene therapy for treatment of Inborn errors of Immunity (IEIs) have
demonstrated significant clinical benefit in multiple Phase I/II clinical trials. Current …

Hematopoietic stem cells (HSCs) are precursor cells that give rise to blood, immune and
tissue-resident progeny in humans. Their position at the starting point of hematopoiesis …

As HSC GT is becoming available for more diseases, novel developments should focus on
improving availability while reducing costs of the treatment. Continued follow up of treated …

Lentiviral transduction of human mesenchymal stem cells (MSCs) induces long-term
transgene expression and holds great promise for multiple gene therapy applications …

Transcriptional enhancers can be in physical proximity of their target genes via chromatin
looping. The enhancer at the β-globin locus (locus control region [LCR]) contacts the fetal …

Genetic modification of cells using viral vectors has shown huge therapeutic benefit in
multiple diseases. However, inefficient transduction contributes to the high cost of these …

Autologous chimeric antigen receptor (CAR) T cells targeting the CD19 antigen have
demonstrated a high complete response rate in relapsed/refractory B-cell malignancies …