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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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Genome Size and the Evolution of New Genes03:21

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While every living organism has a genome of some kind (be it RNA, or DNA), there is considerable variation in the sizes of these blueprints. One major factor that impacts genome size is whether the organism is prokaryotic or eukaryotic. In prokaryotes, the genome contains little to no non-coding sequence, such that genes are tightly clustered in groups or operons sequentially along the chromosome. Conversely, the genes in eukaryotes are punctuated by long stretches of non-coding sequence.
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CRISPR/Cas9 Genome Editing01:28

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Genomics02:02

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Genomics is the science of genomes: it is the study of all the genetic material of an organism. In humans, the genome consists of information carried in 23 pairs of chromosomes in the nucleus, as well as mitochondrial DNA. In genomics, both coding and non-coding DNA is sequenced and analyzed. Genomics allows a better understanding of all living things, their evolution, and their diversity. It has a myriad of uses: for example, to build phylogenetic trees, to improve productivity and...
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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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Gene Therapy and Genome Editing.

Farid Boulad1, Jorge Mansilla-Soto2, Annalisa Cabriolu2

  • 1Center for Cell Engineering, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065, USA; Department of Pediatrics, Memorial Sloan Kettering Cancer Center, 1275 York Avenue, New York, NY 10065, USA.

Hematology/Oncology Clinics of North America
|February 21, 2018
PubMed
Summary

Gene therapy offers a potential cure for severe beta-thalassemia, an inherited blood disorder. Future research focuses on improving lentiviral vectors and conditioning regimens for safer, effective treatments.

Keywords:
CRISPR/Cas9Gene editingGene transferLentivirusThalassemia

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Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Beta-thalassemia comprises over 200 inherited mutations causing reduced beta-globin chain production.
  • Severe forms, like beta-thalassemia major, necessitate lifelong blood transfusions.
  • Current curative options are limited, with allogeneic stem cell transplantation carrying immunologic risks.

Purpose of the Study:

  • To explore the potential of globin gene therapy as a curative treatment for beta-thalassemia.
  • To discuss advancements and future directions in gene therapy for severe hemoglobin disorders.

Main Methods:

  • Review of existing literature on beta-thalassemia and gene therapy approaches.
  • Analysis of globin gene therapy, focusing on lentiviral vector-based strategies.
  • Examination of conditioning regimens and vector safety in the context of autologous stem cell transplantation.

Main Results:

  • Globin gene therapy presents a promising avenue for autologous stem cell transplantation.
  • This approach aims to circumvent the immunological complications associated with allogeneic transplantation.
  • Ongoing research focuses on enhancing lentiviral vectors and optimizing conditioning protocols.

Conclusions:

  • Gene therapy holds significant promise for a curative treatment for severe beta-thalassemia.
  • Advancements in genetic engineering are crucial for developing safer and more effective gene therapy vectors.
  • Future directions include refining lentiviral vector systems and conditioning regimens for improved patient outcomes.