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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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Kidney Transplant I: Introduction01:28

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A kidney transplant is a surgical approach that involves replacing a non-functioning kidney with a healthy one from a donor. This procedure is often a treatment option for end-stage renal disease (ESRD) patients. The method requires careful recipient selection, including evaluating various medical and psychosocial factors. These criteria vary between transplant centers but generally include assessments of the patient's overall health, adherence to medical recommendations, and lifestyle...
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Nephrons01:10

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The kidneys are intricate organs with millions of working units known as nephrons. Each nephron features two major structures: the renal corpuscle, which facilitates blood plasma filtration, and the renal tubule, which handles the glomerular filtrate. Blood supply is directly linked to the nephrons. The renal corpuscle consists of the glomerulus, a capillary network, and the Bowman's capsule, a double-walled epithelial structure that encases the glomerulus. The filtering of blood plasma...
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Kidney Transplant II: Surgical Procedure01:26

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Preoperative ManagementThe primary goals of preoperative management in kidney transplantation are to optimize the patient’s metabolic state and prepare them for surgery through diet adjustments, necessary dialysis, and tailored medical treatment. This phase also involves comprehensive infection screening and patient education about the surgical procedure and postoperative care to improve outcomes and adherence.Medical ManagementA comprehensive evaluation is required for both the living...
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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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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.
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Related Experiment Video

Updated: Jun 9, 2025

Isogenic Kidney Glomerulus Chip Engineered from Human Induced Pluripotent Stem Cells
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Gene therapy and kidney diseases.

Nadia Tavakolidakhrabadi1, Wen Y Ding1, Moin A Saleem1,2

  • 1Bristol Renal, University of Bristol, Dorothy Hodgkin Building, Whitson Street, Bristol BS1 3NY, UK.

Molecular Therapy. Methods & Clinical Development
|October 22, 2024
PubMed
Summary

Gene therapy offers a promising new approach for chronic kidney disease (CKD), targeting disease-causing genes with advanced delivery systems for improved kidney health.

Keywords:
gene therapykidney diseasenanodelivery systemstargeted gene deliveryviral vectors

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Area of Science:

  • Nephrology
  • Genetics
  • Biotechnology

Background:

  • Chronic kidney disease (CKD) is a major global health issue, predicted to be a leading cause of death by 2040.
  • Existing treatments for CKD focus on managing symptoms and slowing disease progression, not addressing underlying genetic causes.
  • Advances in understanding kidney disease genomics and cell-specific gene therapy have created new therapeutic possibilities.

Purpose of the Study:

  • To review the potential of gene therapy and genome editing for treating kidney diseases.
  • To explore various delivery routes and systems for targeting specific kidney cells.
  • To discuss how gene therapy can be applied to enhance kidney health.

Main Methods:

  • Review of current scientific literature on gene therapy and genome editing in the context of kidney diseases.
  • Analysis of different delivery strategies for targeting renal cells.
  • Examination of the application of gene therapy for improving kidney function and health.

Main Results:

  • Gene therapy and genome editing show significant promise as personalized treatments for kidney diseases.
  • Various delivery systems are being developed to effectively target specific kidney cells.
  • Successful application of these technologies could revolutionize kidney disease management.

Conclusions:

  • Targeting disease-causing genes through gene therapy represents a paradigm shift in treating chronic kidney disease.
  • Further research into efficient and safe delivery methods is crucial for clinical translation.
  • Gene therapy holds the potential to offer curative or significantly improved outcomes for patients with kidney disease.