The first therapeutic nucleic acid, a DNA oligonucleotide, was approved for clinical use in
1998. Twenty years later, in 2018, the first therapeutic RNA-based oligonucleotide was …

Nucleic acid aptamers, often termed'chemical antibodies', are functionally comparable to
traditional antibodies, but offer several advantages, including their relatively small physical …

Background CCR5 is the major coreceptor for human immunodeficiency virus (HIV). We
investigated whether site-specific modification of the gene (“gene editing”)—in this case, the …

Antiretroviral therapy is not curative. Given the challenges in providing lifelong therapy to a
global population of more than 35 million people living with HIV, there is intense interest in …

Background Gene delivery systems are essentially necessary for the gene therapy of human
genetic diseases. Gene therapy is the unique way that is able to use the adjustable gene to …

Abstract Recently, United States Food and Drug Administration (FDA) and European
Commission (EC) approved Alnylam Pharmaceuticals' RNA interference (RNAi) therapeutic …

Recent advances of biological drugs have broadened the scope of therapeutic targets for a
variety of human diseases. This holds true for dozens of RNA-based therapeutics currently …

Cell therapies have emerged as a promising therapeutic modality with the potential to treat
and even cure a diverse array of diseases. Cell therapies offer unique clinical and …

Improvements in the gene transfer vectors used in therapeutic trials have led to substantial
clinical successes in patients with serious genetic conditions, such as immunodeficiency …

RNA interference (RNAi) is a powerful approach for reducing expression of endogenously
expressed proteins. It is widely used for biological applications and is being harnessed to …