Hematological disorders result in significant health
consequences,
and traditional therapies frequently entail adverse reactions without
addressing the root cause. A potential solution for hematological
disorders characterized by gain-of-function mutations lies in the
emergence of small interfering RNA (siRNA) molecules as a therapeutic
option. siRNAs are a class of RNA molecules composed of double-stranded
RNAs that can degrade specific mRNAs, thereby inhibiting the synthesis
of underlying disease proteins. Therapeutic interventions utilizing
siRNA can be tailored to selectively target genes implicated in diverse
hematological disorders, including sickle cell anemia, β-thalassemia,
and malignancies such as lymphoma, myeloma, and leukemia. The development
of efficient siRNA silencers necessitates meticulous contemplation
of variables such as the RNA backbone, stability, and specificity.
Transportation of siRNA to specific cells poses a significant hurdle,
prompting investigations of diverse delivery approaches, including
chemically modified forms of siRNA and nanoparticle formulations with
various biocompatible carriers. This review delves into the crucial
role of siRNA technology in targeting and treating hematological malignancies
and disorders. It sheds light on the latest research, development,
and clinical trials, detailing how various pharmaceutical approaches
leverage siRNA against blood disorders, mainly concentrating on cancers.
It outlines the preferred molecular targets and physiological barriers
to delivery while emphasizing the growing potential of various therapeutic
delivery methods. The need for further research is articulated in
the context of overcoming the shortcomings of siRNA in order to enrich
discussions around siRNA’s role in managing blood disorders
and aiding the scientific community in advancing more targeted and
effective treatments.
