It is now well known that non-coding RNAs (ncRNAs), rather than protein-coding transcripts,
are the preponderant RNA transcripts. NcRNAs, particularly microRNAs (miRNAs), long non …

Recent advances in the generation, purification and cellular delivery of RNA have enabled
development of RNA-based therapeutics for a broad array of applications. RNA therapeutics …

More than two decades after the natural gene-silencing mechanism of RNA interference was
elucidated, small interfering RNA (siRNA)-based therapeutics have finally broken into the …

Five small interfering RNA (siRNA)-based therapeutics have been approved by the Food
and Drug Administration (FDA), namely patisiran, givosiran, lumasiran, inclisiran, and …

Transthyretin (TTR) is a tetrameric protein synthesized mostly by the liver and secreted into
the plasma. TTR molecules can misfold and form amyloid fibrils in the heart and peripheral …

Triple-negative breast cancer (TNBC) cells are deficient in estrogen, progesterone and
ERBB2 receptor expression, presenting a particularly challenging therapeutic target due to …

In the realm of gene therapy, a pivotal moment arrived with Paul Berg's groundbreaking
identification of the first recombinant DNA in 1972. This achievement set the stage for future …

Hyaluronic acid is a naturally occurring biopolymer possessing numerous functions within
the body including wound repair, cell migration, and cell signaling. Due to its versatility …

Over the past decade, the increased adoption of electroporation-based technologies has led
to an expansion of clinical research initiatives. Electroporation has been utilized in …

Inherited retinal diseases (IRDs) are a clinically complex and heterogenous group of visual
impairment phenotypes caused by pathogenic variants in at least 277 nuclear and …