Abstract
Aptamers are RNA/DNA oligonucleotide molecules that specifically bind to a targeted complementary molecule. As potential recognition elements with promising diagnostic and therapeutic applications, aptamers, such as monoclonal antibodies, could provide many treatment and diagnostic options for blood diseases. Aptamers present several superior features over antibodies, including a simple in vitro selection and production, ease of modification and conjugation, high stability, and low immunogenicity. Emerging as promising alternatives to antibodies, aptamers could overcome the present limitations of monoclonal antibody therapy to provide novel diagnostic, therapeutic, and preventive treatments for blood diseases. Researchers in several biomedical areas, such as biomarker detection, diagnosis, imaging, and targeted therapy, have widely investigated aptamers, and several aptamers have been developed over the past two decades. One of these is the pegaptanib sodium injection, an aptamer-based therapeutic that functions as an anti-angiogenic medicine, and it is the first aptamer approved by the U.S. Food and Drug Administration (FDA) for therapeutic use. Several other aptamers are now in clinical trials. In this review, we highlight the current state of aptamers in the clinical trial program and introduce some promising aptamers currently in pre-clinical development for blood diseases.
Highlights
Nucleic acid aptamers constitute a special class of synthetic polymers or oligomers of single-stranded ssDNA or RNA molecules, and they have the capacity to bind to a specific target by forming secondary and/or tertiary structures
The SELEX procedure was used for the aptamer selection process, and in a typical SELEX, it was carried out using purified target molecules, starting with a large library of random oligonucleotides
Aptamers offer a set of tools for novel diagnostics, drug delivery, and therapeutics to treat several types of diseases
Summary
Nucleic acid aptamers constitute a special class of synthetic polymers or oligomers of single-stranded ssDNA or RNA molecules, and they have the capacity to bind to a specific target by forming secondary and/or tertiary structures. Short half-life in (susceptible to serum degradation and and susceptible renal filtration) renal to filtration). (b) SELEX technology selects specific aptamers from random DNA, RNA, or peptide with an antigen that provokes an immune response for mAb production. This approach presents libraries without sacrificing animals. Aptamers are interies withoutbe sacrificing mediate in size (8–15 kDa) between antibodies (150 kDa) and small peptides (1–5 kDa), Aptamers can bind to highly toxic or non-immunogenic antigens, an attribute that cannot be achieved with animal-based methods of mAb production. Because aptamer-based therapeutic properties are similar to those of mAbs, aptamers can be described as chemical antibodies They are capable of binding and inhibiting the immunoregulatory components of carcinogenesis, and they can be used as carriers for therapeutic agents. We focus on aptamer applications in the diagnosis and therapy of hematological diseases [6,7]
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