A resurgence in clinical trials using RNA interference (RNAi) occurred in 2012. Although there
were initial difficulties in achieving efficacious results with RNAi without toxic side effects, …

Aptamers are single-stranded nucleic acids that specifically recognize and bind tightly to
their cognate targets due to their stable three-dimensional structure. Nucleic acid aptamers …

HIV/AIDS is a chronic and debilitating disease that cannot be cured with current antiretroviral
drugs. While combinatorial antiretroviral therapy (cART) can potently suppress HIV-1 …

CRISPR–Cas genome-editing nucleases hold substantial promise for developing human
therapeutic applications 1 , 2 , 3 , 4 , 5 – 6 but identifying unwanted off-target mutations is …

CRISPR-Cas genome-editing nucleases hold substantial promise for human therapeutics 1-5
but 34 identifying unwanted off-target mutations remains an important requirement for …

Recently, there has been significant interest in the use of mRNA based expression systems
for gene therapy applications and for the generation and manipulation of stem cells. Several …

Background Chimeric antigen receptor (CAR)-T cell therapies for the treatment of hematological
malignancies experienced tremendous progress in the last decade. However, essential …

Background Adoptive transfer of chimeric antigen receptor (CAR)-engineered T cells combined
with checkpoint inhibition may prevent T cell exhaustion and improve clinical outcomes. …

Background Interleukin (IL)-12 activates T cells and macrophages pivoting the switch that
turns chronic into acute inflammation and results in cancer rejection. However, despite …

Background The application of CRISPR-Cas9 for personalized medicine is potentially
revolutionary for the treatment of several diseases including cancer. However, the bacterial origin …