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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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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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Histone Modification02:32

Histone Modification

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The histone proteins have a flexible N-terminal tail extending out from the nucleosome. These histone tails are often subjected to post-translational modifications such as acetylation, methylation, phosphorylation, and ubiquitination. Particular combinations of these modifications form “histone codes” that influence the chromatin folding and tissue-specific gene expression.
Acetylation
The enzyme histone acetyltransferase adds acetyl group to the histones. Another enzyme, histone...
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Hypertension IV: Drug Therapy and Lifestyle Modifications01:28

Hypertension IV: Drug Therapy and Lifestyle Modifications

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Multiple classes of antihypertensive medications are employed in treating hypertension. The most commonly recommended first-line treatments include:Thiazide Diuretics, such as chlorthalidone, increase sodium and water excretion from the body, reducing blood volume and blood pressure.Angiotensin-converting enzyme inhibitors, like lisinopril, block the conversion of angiotensin I to II, a potent vasoconstrictor lowering blood pressure.Angiotensin II Receptor Blockers (ARBs) prevent angiotensin II...
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Spreading of Chromatin Modifications02:25

Spreading of Chromatin Modifications

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The histone proteins in the nucleosomes are post-translationally modified (PTM) to increase or decrease access to DNA. The commonly observed PTMs are methylation, acetylation, phosphorylation, and ubiquitination of lysine amino acids in the histone H3 tail region. These histone modifications have specific meaning for the cell. Hence, they are called "histone code". The protein complex involved in histone modification is termed as "reader-writer" complex.
Writers
The writer...
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Export of Mitochondrial and Chloroplast Genes02:19

Export of Mitochondrial and Chloroplast Genes

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A eukaryotic cell can have up to three different types of genetic systems: nuclear, mitochondrial, and chloroplast. During evolution, organelles have exported many genes to the nucleus; this transfer is still ongoing in some plant species. Approximately 18% of the Arabidopsis thaliana nuclear genome is thought to be derived from the chloroplast’s cyanobacterial ancestor, and around 75% of the yeast genome derived from the mitochondria’s bacterial ancestor. This export has occurred...
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Updated: Feb 14, 2026

Combined Genetic and Chemical Capsid Modifications of Adenovirus-Based Gene Transfer Vectors for Shielding and Targeting
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Development of Targeted Therapies Based on Gene Modification.

Taylor M Benson1, Fatjon Leti1, Johanna K DiStefano2

  • 1Department of Biomedical Research, Center for Genes, Environment, and Health, National Jewish Health, 1400 Jackson Street, Denver, CO, 80206, USA.

Methods in Molecular Biology (Clifton, N.J.)
|February 10, 2018
PubMed
Summary

Precision medicine and gene therapy leverage next-generation sequencing to identify disease-causing genes. This enables targeted treatments, moving beyond generalized symptom management for improved healthcare.

Keywords:
Gene therapyNGS, GWASPersonalized medicinePharmacogenomicsPrecision medicine

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Area of Science:

  • Genomics
  • Biotechnology
  • Medical Genetics

Background:

  • Next-generation sequencing (NGS) drives advancements in personalized healthcare.
  • Precision medicine aims to tailor treatments based on individual genetic makeup.
  • Current approaches often involve generalized symptom treatment, lacking personalization.

Purpose of the Study:

  • To introduce precision medicine and its importance in disease treatment.
  • To highlight the role of identifying genetic variants in disease pathogenesis.
  • To discuss current therapeutic strategies and future challenges in precision medicine.

Main Methods:

  • Review of current literature on precision medicine and gene therapy.
  • Discussion of gene identification techniques and their application.
  • Exploration of targeted therapeutic approaches based on genetic data.

Main Results:

  • Identification of disease-associated genes is crucial for precision medicine.
  • Targeted therapies offer a personalized alternative to traditional treatments.
  • Understanding genetic contributions enhances disease pathology comprehension.

Conclusions:

  • Precision medicine, supported by genetic insights, is poised to become a new clinical standard.
  • Further research into gene-disease relationships is essential for therapeutic development.
  • Addressing challenges in precision medicine will optimize its clinical implementation.