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RNA Editing02:23

RNA Editing

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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...
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Uncertainty in Measurement: Accuracy and Precision03:37

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Scientists typically make repeated measurements of a quantity to ensure the quality of their findings and to evaluate both the precision and the accuracy of their results. Measurements are said to be precise if they yield very similar results when repeated in the same manner. A measurement is considered accurate if it yields a result that is very close to the true or the accepted value. Precise values agree with each other; accurate values agree with a true value. 
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Accuracy and Precision01:52

Accuracy and Precision

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Scientists typically make repeated measurements of a quantity to ensure the quality of their findings and to evaluate both the precision and the accuracy of their results. Measurements are said to be precise if they yield very similar results when repeated in the same manner. A measurement is considered accurate if it yields a result that is very close to the true or the accepted value. Precise values agree with each other; accurate values agree with a true value.  Highly accurate...
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Gene Therapy00:59

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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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Extraction: Advanced Methods00:56

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Metal ions can be separated from one another by complexation with organic ligands–the chelating agent– to form uncharged chelates. Here, the chelating agent must contain hydrophobic groups and behave as a weak acid, losing a proton to bind with the metal. Since most organic ligands used in this process are insoluble or undergo oxidation in the aqueous phase, the chelating agent is initially added to the organic phase and extracted into the aqueous phase. The metal-ligand complex is...
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Overview of Advanced Functional Groups02:22

Overview of Advanced Functional Groups

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Functional groups are groups of atoms with specific chemical properties that occur within organic molecules and are sometimes denoted as “R”. Functional groups can “functionalize” a compound by enabling it to adopt different physical and chemical properties.
Types of Advanced Functional Groups
The table below summarizes some of the major functional groups in organic chemistry.
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CRISPR/Cas9 Ribonucleoprotein-mediated Precise Gene Editing by Tube Electroporation
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Advances in Precision Editing Therapies for Alpha-1 Antitrypsin Deficiency.

Jenny Gao1, Erik Sontheimer1,2,3,4, Terence R Flotte3,5,6

  • 1RNA Therapeutics Institute, University of Massachusetts Chan Medical School, Worcester, Massachusetts, USA.

Human Gene Therapy
|January 27, 2026
PubMed
Summary

Genome and RNA editing offer safer gene therapy alternatives. This review focuses on precision medicine approaches for Alpha-1 antitrypsin deficiency (AATD), examining current preclinical and clinical editing strategies for this genetic disorder.

Keywords:
RNA editingalpha-1 antitrypsinbase editinggene editingprime editing

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Area of Science:

  • Biotechnology
  • Genetics
  • Precision Medicine

Background:

  • Alpha-1 antitrypsin deficiency (AATD) is a genetic disorder caused by mutations in a single gene, leading to liver and lung diseases.
  • The well-defined disease mechanism makes AATD a prime candidate for precision medicine interventions.
  • Traditional gene replacement therapies have limitations, driving the need for alternative approaches.

Purpose of the Study:

  • To review current preclinical and clinical research on Alpha-1 antitrypsin deficiency (AATD).
  • To highlight the application of genome and RNA editing strategies for AATD.
  • To provide an overview of precision medicine advancements for AATD.

Main Methods:

  • Literature review of preclinical studies on AATD.
  • Analysis of ongoing clinical trials for AATD treatments.
  • Focus on genome editing techniques (e.g., CRISPR-Cas9) and RNA editing approaches.

Main Results:

  • Genome and RNA editing show promise as safer alternatives to gene replacement.
  • Preclinical data suggest feasibility of editing strategies for AATD.
  • Clinical efforts are underway to translate these editing technologies into effective therapies.

Conclusions:

  • Genome and RNA editing represent a significant advancement in precision gene therapy for AATD.
  • These editing modalities offer a targeted approach to address the genetic basis of AATD.
  • Continued research and clinical development are crucial for realizing the therapeutic potential of gene editing in AATD.