Related Experiment Video
Updated: Jul 17, 2026
11:34
Exploring Sequence Space to Identify Binding Sites for Regulatory RNA-Binding Proteins
Published on: August 9, 2019
Gene therapy progress and prospects: RNA aptamers
N S Que-Gewirth1, B A Sullenger
1Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.
Gene Therapy
|February 7, 2007
Summary
Aptamers are DNA or RNA molecules that bind targets with high specificity. Their safety, efficacy, and reversibility make them promising therapeutic agents for various diseases.
Area of Science:
- Biochemistry
- Molecular Biology
- Drug Discovery
Background:
- Aptamers are nucleic acid-based ligands with high affinity and specificity for target molecules.
- They offer advantages over traditional therapeutics, including stability, low toxicity, and reduced immunogenicity.
Purpose of the Study:
- To highlight the growing importance of aptamers in therapeutics, drug development, and diagnostics.
- To emphasize the potential of aptamers as safe and effective therapeutic agents.
Main Methods:
- In vitro evolution (SELEX) for aptamer selection.
- Characterization of aptamer binding affinity and specificity.
- Evaluation of aptamer stability, toxicity, and immunogenicity.
Main Results:
- Aptamers demonstrate high affinity and specificity for diverse molecular targets.
- Therapeutic aptamers exhibit favorable safety profiles and stability.
- Reversibility of aptamer activity with an antidote enhances therapeutic potential.
Conclusions:
- Aptamers represent a promising class of therapeutic agents with broad applicability.
- The development of aptamer-based drugs is rapidly advancing, supported by regulatory approvals and innovative strategies like antidote systems.
Related Concept Videos
00:59Gene Therapy
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
00:59Gene Therapy
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
01:29Microorganisms in Medicine and Therapeutics
Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
02:23RNA Editing
RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
02:15Experimental RNAi
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
01:29Pharmacogenomics: Identification of New Drug Targets
Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...