Extracellular vesicles (EVs) are submicron cell-secreted structures containing proteins,
nucleic acids and lipids. EVs can functionally transfer these cargoes from one cell to another …

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 …

Delivering protein therapeutics specifically into target cells and tissues is a promising
avenue in medicine. Advancing this process will significantly enhance the efficiency of the …

MicroRNAs play an essential role in cell homeostasis and have been proposed as
therapeutic agents. One strategy to deliver microRNAs is to genetically engineer target cells …

Viral vectors are an emerging, exciting class of biologics whose application in vaccines,
oncology, and gene therapy has grown exponentially in recent years. Following first …

Dent disease type 1 is caused by changes in the chloride voltage-gated channel 5 (CLCN5)
gene on chromosome X, resulting in the lack or dysfunction of chloride channel ClC-5 …

Autoimmune destruction of pancreatic beta cells is the characteristic feature of type 1
diabetes mellitus. Consequently, both short-and intermediate-acting insulin analogs are …

Over the past 3 decades, there has been significant progress in refining gene therapy
technologies and procedures. Transduction of hematopoietic stem cells ex vivo using …

Hematopoietic stem and progenitor cell (HSPC)-based ex vivo gene therapy has
demonstrated clinical success for X-linked severe combined immunodeficiency (SCID-X1) …

Abstract Interleukin-6 (IL-6) is a highly pro-inflammatory cytokine involved in the
etiopathology of several inflammatory diseases and cancer. As so, the inhibition of IL-6 …