MicroRNA (miRNA) is regarded as a prominent genetic regulator, as it can fine‐tune an
entire biological pathway by targeting multiple target genes. This characteristic makes …

In recent years, nanoparticles derived from cellular membranes have been increasingly
explored for the prevention and treatment of human disease. With their flexible design and …

Recently, clinical trials of adeno-associated virus-mediated replacement therapy have
suggested long-term therapeutic effects for several genetic diseases of the liver, including …

Gene therapies, despite of being a relatively new therapeutic approach, have a potential to
become an important alternative to current treatment strategies in glaucoma. Since …

HIV-1 derived lentiviral vector is an efficient transporter for delivering desired genetic
materials into the targeted cells among many viral vectors. Genetic material transduced by …

In the current phase of medical progress, practical neuro-oncology faces critical challenges.
These include the quest for and development of innovative methodological approaches, as …

Hematopoietic stem cell (HSC) mutations can result in clonal hematopoiesis (CH) with
heterogeneous clinical outcomes. Here, we investigate how the cell state preceding Tet2 …

Gene therapy in pediatrics represents a cutting-edge therapeutic strategy for treating a
range of genetic disorders that manifest in childhood. Gene therapy involves the …

The field of retroviral integration research has a long history that started with the provirus
hypothesis and subsequent discoveries of the retroviral reverse transcriptase and integrase …

Deficiency of adenosine deaminase type 2 (DADA2) is an autosomal recessive disease
caused by bi-allelic loss-of-function mutations in ADA2. Treatment with anti-TNF is effective …