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Related Concept Videos

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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FDA Approved Drugs: Changes to Approved Drugs01:26

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Post-approval, manufacturers may modify an approved new or generic drug product. Such modifications can encompass alterations in the Active Pharmaceutical Ingredient (API), manufacturing process, formulation, batch size, manufacturing site, and container closure system (FDA Guidance for Industry, April 2004). Often, a drug product may undergo multiple changes.These modifications require careful evaluation to determine their potential impact on the drug product's identity, strength, quality,...
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The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
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A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells
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Oligonucleotides to the (Gene) Rescue: FDA Approvals 2017-2019.

Jacqueline Rüger1, Silvia Ioannou2, Daniela Castanotto1

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Oligonucleotide therapeutics, including siRNA, show renewed promise with recent FDA approvals after years of limited success. Challenges remain regarding efficacy, toxicity, and high costs impacting accessibility.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Oligonucleotides have been explored for gene expression manipulation for 40 years.
  • Early FDA approvals (pre-2016) yielded drugs with poor market performance.
  • The clinical potential of small interfering RNA (siRNA) therapeutics had diminished over 15 years.

Purpose of the Study:

  • To review the field of oligonucleotide therapeutics.
  • To provide an update on FDA approvals of oligonucleotide drugs from 2017 to Q2 2019.
  • To consider ethical issues, efficacy, toxicity, and cost of these therapies.

Main Methods:

  • Literature review of oligonucleotide therapeutics.
  • Analysis of FDA approval data for oligonucleotides (2017-Q2 2019).
  • Discussion of clinical and ethical considerations.

Main Results:

  • Several new FDA approvals for oligonucleotide therapeutics have occurred in the last 4 years (2017-Q2 2019).
  • This recent activity suggests a resurgence in the field.
  • Despite progress, challenges persist.

Conclusions:

  • A new era for oligonucleotide and siRNA clinical therapeutics is emerging.
  • Limited efficacy, treatment toxicity, and high costs remain significant challenges.
  • Accessibility of these advanced therapies is a major concern.