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Related Concept Videos

Next-generation Sequencing03:00

Next-generation Sequencing

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The first human genome sequencing project cost $2.7 billion and was declared complete in 2003, after 15 years of international cooperation and collaboration between several research teams and funding agencies. Today, with the advent of next-generation sequencing technologies, the cost and time of sequencing a human genome have dropped over 100 fold.
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Nucleic Acids02:43

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Nucleic Acids and Nucleotides01:20

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Nucleic acids are the most important macromolecules for the continuity of life. They carry the cell's genetic blueprint and have instructions for its functioning. The two main types of nucleic acids are deoxyribonucleic acid (DNA) and ribonucleic acid (RNA).
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Gene Therapy00:59

Gene Therapy

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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...
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Nucleic Acid Structure01:25

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The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
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Sanger Sequencing01:57

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DNA sequencing is a fundamental technique that is routinely used in the biological sciences. This method can be applied to a range of questions at different scales - from the sequencing of a cloned DNA fragment or the study of a mutation in a gene up to whole-genome sequencing. However, despite the widespread use of sequencing today, it was not until 1977 that Fredrick Sanger and his collaborators developed the chain-termination method to decode DNA sequences. It relies on the separation of a...
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Updated: May 29, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids
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Nucleic acid therapeutics: Past, present, and future.

Sajid Naeem1,2, Ju Zhang1,2, Yang Zhang3

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Molecular Therapy. Nucleic Acids
|February 3, 2025
PubMed
Summary

Nucleic acid therapeutics, including siRNA and mRNA, offer new ways to treat diseases by targeting genetic sequences. Advances in engineering and delivery are overcoming challenges for wider therapeutic use.

Keywords:
COVID-19 vaccinesMT: Oligonucleotides: Therapies and ApplicationsN-acetylgalactosamine conjugatesantisense oligonucleotidesaptamerlipid nanoparticlesmessenger RNAnucleic acid therapeuticssmall interfering RNA

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Pharmacology

Background:

  • Nucleic acid therapeutics target coding and non-coding sequences.
  • Approved modalities include siRNA, mRNA, aptamers, and antisense oligonucleotides.
  • COVID-19 vaccines and CRISPR-Cas9 have accelerated the field.

Purpose of the Study:

  • To provide an overview of nucleic acid therapeutics.
  • To highlight advancements in engineering, conjugation, and delivery strategies.
  • To discuss the growing role of nucleic acid therapeutics in medicine.

Main Methods:

  • Review of current literature on nucleic acid therapeutics.
  • Analysis of engineering, conjugation, and delivery strategies.
  • Discussion of regulatory approvals and clinical applications.

Main Results:

  • Several nucleic acid modalities are approved for therapeutic use.
  • Significant progress has been made in overcoming delivery challenges.
  • CRISPR-Cas9 represents a major advancement.

Conclusions:

  • Nucleic acid therapeutics are a rapidly growing area with significant potential.
  • Engineering and delivery innovations are crucial for clinical success.
  • These therapies are poised to play a larger role in modern medicine.