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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.4K
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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Genomic Imprinting and Inheritance02:30

Genomic Imprinting and Inheritance

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Diploid organisms inherit genetic material through chromosomes from both parents. Copies of the same gene are known as alleles. In most cases, both alleles are simultaneously expressed and allow various cellular processes to function optimally. If one of the alleles is missing or mutated, the expression of the other allele can compensate; however, this is not true for all genes.
The expression of some genes depends on which parent passed the gene to the offspring, through a phenomenon known as...
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Chromosomal Theory of Inheritance01:39

Chromosomal Theory of Inheritance

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In 1866, Gregor Mendel published the results of his pea plant breeding experiments, providing evidence for predictable patterns in the inheritance of physical characteristics. The significance of his findings was not immediately recognized. In fact, the existence of genes was unknown at the time. Mendel referred to hereditary units as “factors.”
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Inheritance of Chromatin Structures03:17

Inheritance of Chromatin Structures

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Epigenetics is the study of inherited changes in a cell's phenotype without changing the DNA sequences. It provides a form of memory for the differential gene expression pattern to maintain cell lineage, position-effect variegation, dosage compensation, and maintenance of chromatin structures such as telomeres and centromeres. For example, the structure and location of the centromere on chromosomes are epigenetically inherited. Its functionality is not dictated or ensured by the underlying...
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Non-nuclear Inheritance01:29

Non-nuclear Inheritance

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Most DNA resides in the nucleus of a cell. However, some organelles in the cell cytoplasm⁠—such as chloroplasts and mitochondria⁠—also have their own DNA. These organelles replicate their DNA independently of the nuclear DNA of the cell in which they reside. Non-nuclear inheritance describes the inheritance of genes from structures other than the nucleus.
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Inheritance01:25

Inheritance

1.5K
Gregor Mendel's pioneering work on the principles of inheritance fundamentally transformed our understanding of how traits are transmitted from generation to generation. His experiments with pea plants laid the groundwork for the discovery of genes, discrete units within organisms that control heredity.
Each gene exists in pairs, and the combination of these genes from both parents forms an individual's genotype. This genotype is a blueprint of potential traits. Examples of genotype...
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Related Experiment Video

Updated: Jan 22, 2026

Cortical Neurogenesis: Transitioning from Advances in the Laboratory to Cell-Based Therapies
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Advances in Gene Therapy for Inherited Haemoglobinopathies.

Anna B Gaspar1, H Bobby Gaspar2,3

  • 1Kings College Hospital Foundation Trust, London SE5 9RS, UK.

Hematology Reports
|January 21, 2026
PubMed
Summary

Gene therapy offers a curative approach for haemoglobinopathies like sickle cell disease (SCD) and β-thalassaemia, advancing beyond supportive care and traditional stem cell transplants. Current gene therapies show promise but face challenges in accessibility and cost.

Keywords:
gene editinggene therapyinherited haemoglobinopathiessickle cell diseasethalassaemia

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Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Haemoglobinopathies, including β-thalassaemia and sickle cell disease (SCD), are common monogenic disorders causing significant morbidity and mortality.
  • Traditional management relies on supportive care, while allogeneic haematopoietic stem cell transplantation (HSCT) is curative but high-risk and limited in application.

Purpose of the Study:

  • To review the progression and current status of gene therapy for haemoglobinopathies.
  • To highlight the success of gene addition, gene silencing, and gene editing strategies.
  • To discuss the challenges and future directions for accessible gene therapy.

Main Methods:

  • Review of lentiviral gene addition for β-like globin expression.
  • Analysis of gene silencing strategies targeting BCL11A.
  • Evaluation of gene editing technologies like CRISPR/Cas9 and base editing.

Main Results:

  • Gene addition therapies have achieved transfusion independence in β-thalassaemia and reduced complications in SCD.
  • BCL11A gene silencing and CRISPR/Cas9 gene editing have shown clinical success, leading to approved therapies.
  • Emerging base editing technologies show potential for precise genetic modification.

Conclusions:

  • Gene therapy has rapidly evolved into an approved and effective treatment for haemoglobinopathies.
  • Despite advances, challenges in manufacturing, conditioning regimens, and cost limit accessibility.
  • Further improvements are crucial for gene therapy to become a widely accessible, curative option.