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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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Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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What is Gene Expression?01:42

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Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
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Cell-Based Gene Therapy System for Delivering BMPs.

Austin Dickerson1, Eleanor L Davis1, Corinne Sonnet1

  • 1Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital and Houston Methodist Hospital, Houston, TX, USA.

Methods in Molecular Biology (Clifton, N.J.)
|November 11, 2018
PubMed
Summary
This summary is machine-generated.

This study introduces an adenoviral vector system for in vivo protein generation. The method uses transduced cells to produce bone morphogenetic protein 2 (BMP-2), effectively stimulating new bone formation.

Keywords:
Adenoviral transductionAlkaline phosphatase assayBone formationBone morphogenetic proteinCell-based gene therapyDelivery systemOsteoinduction

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Area of Science:

  • Biotechnology
  • Regenerative Medicine
  • Molecular Biology

Background:

  • Adenoviral vectors enable in vivo protein production with accurate posttranslational processing.
  • Transduced cells offer advantages over direct viral delivery, including reduced toxicity and controllable expression.

Purpose of the Study:

  • To describe a novel delivery system for in vivo generation of therapeutic proteins.
  • To utilize adenoviral transduction for targeted delivery of bone morphogenetic protein 2 (BMP-2).

Main Methods:

  • Engineering a non-replicating adenoviral vector containing BMP-2 in the E1 region.
  • Transducing eukaryotic cells with the engineered adenoviral vector.
  • Utilizing transduced cells for localized protein expression and therapeutic effect.

Main Results:

  • Demonstrated successful in vivo generation of secreted proteins via transduced cells.
  • Showcased the ability of small amounts of BMP-2 to induce de novo bone formation.
  • Highlighted the controllable and targeted nature of the therapeutic approach.

Conclusions:

  • The described adenoviral vector system provides a safe and effective method for in vivo protein production.
  • This approach facilitates targeted regeneration, specifically promoting new bone formation.
  • The system offers a controllable therapeutic strategy by leveraging cell clearance mechanisms.