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Related Concept Videos

Gene Therapy00:59

Gene Therapy

25.3K
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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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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Human Genetics01:28

Human Genetics

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Human genetics provides a profound framework for understanding the interplay between genetic predispositions and human psychology. At the heart of this discipline lies the study of how genes influence physical traits, behaviors, and susceptibility to diseases. Each person carries a unique genetic code that subtly or significantly shapes their psychological and behavioral landscape.
The complex relationship between genetics and psychology is observable through common biological components such...
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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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Genome-wide Association Studies-GWAS01:11

Genome-wide Association Studies-GWAS

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Genome-wide association studies or GWAS are used to identify whether common SNPs are associated with certain diseases. Suppose specific SNPs are more frequently observed in individuals with a particular disease than those without the disease. In that case, those SNPs are said to be associated with the disease. Chi-square analysis is performed to check the probability of the allele likely to be associated with the disease.
GWAS does not require the identification of the target gene involved in...
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Incomplete Dominance01:43

Incomplete Dominance

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Gregor Mendel's work (1822 - 1884) was primarily focused on pea plants. Through his initial experiments, he determined that every gene in a diploid cell has two variants called alleles inherited from each parent. He suggested that amongst these two alleles, one allele is dominant in character and the other recessive. The combination of alleles determines the phenotype of a gene in an organism.
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Updated: Jun 14, 2025

Electroporation-Based Genetic Modification of Primary Human Pigment Epithelial Cells Using the Sleeping Beauty Transposon System
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Gene therapy for polygenic or complex diseases.

Tingting Wu1,2, Yu Hu3,4, Liang V Tang5,6

  • 1Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, 430022, China.

Biomarker Research
|September 5, 2024
PubMed
Summary
This summary is machine-generated.

Gene therapy offers novel treatments for genetic disorders using nucleic acid drugs. This review explores gene editing and delivery methods, focusing on applications for complex diseases in clinical trials.

Keywords:
CRISPR-CAS9Complex diseasesGene therapyPolygenic diseases

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Candidate Gene Testing in Clinical Cohort Studies with Multiplexed Genotyping and Mass Spectrometry
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Candidate Gene Testing in Clinical Cohort Studies with Multiplexed Genotyping and Mass Spectrometry

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Area of Science:

  • Biotechnology
  • Genetics
  • Pharmacology

Background:

  • Gene therapy, using nucleic acid drugs, provides distinct therapeutic strategies for genetic disorders, differing from traditional medications.
  • Significant advancements in gene therapy over two decades have led to approved drugs, initially for monogenic orphan diseases and cancers.
  • Polygenic diseases, with higher incidence, present new therapeutic targets due to extensive research into their etiology.

Purpose of the Study:

  • To review gene therapy strategies, gene editing techniques (primarily CRISPR-CAS9), and delivery vectors.
  • To highlight current and emerging applications of gene therapy in polygenic or complex diseases.
  • To focus on gene therapy applications that have reached or are undergoing clinical trials.

Main Methods:

  • Review of scientific literature on gene therapy strategies, gene editing technologies, and delivery systems.
  • Analysis of clinical trial data for gene therapy applications in monogenic and polygenic diseases.
  • Focus on CRISPR-CAS9 as a key gene editing method and various viral and non-viral vectors for gene delivery.

Main Results:

  • Gene therapy has evolved from treating rare monogenic diseases to exploring complex polygenic conditions.
  • CRISPR-CAS9 and advanced delivery vectors are crucial for expanding gene therapy applications.
  • Several gene therapy applications for complex diseases are progressing through clinical trials.

Conclusions:

  • Gene therapy holds significant promise for treating a wider range of diseases, including complex polygenic disorders.
  • Continued research and clinical trials are essential to realize the full potential of gene therapy for diverse patient populations.
  • The integration of gene editing and effective delivery systems is key to advancing gene therapy's therapeutic impact.