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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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Gene Therapy00:59

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Clinical Applications of Epidermal Stem Cells01:19

Clinical Applications of Epidermal Stem Cells

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Epidermal stem cells (EpiSCs) are mainly located at the basal layer of the epidermis. These cells repair minor injuries of the skin and replace dead skin cells. However, EpiSCs’ cannot heal severe wounds such as major burns or those from diabetes or hereditary disorders. In such cases, culturing the epidermal stem cells from the patient is possible and has yielded successful treatment options, such as laboratory-grown skin grafts. These grafts are synthesized using a patient’s own...
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Skin Diseases and Disorders01:23

Skin Diseases and Disorders

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Skin is the first line of defense and encounters a variety of microbes. Some pathogenic strains are often the cause of a broad range of infections of the skin and other body systems. These conditions can affect people of all ages and may have different causes, including genetic factors, infections, autoimmune reactions, environmental factors, and lifestyle choices.
Gram-positive Staphylococcus spp. and Streptococcus spp. are responsible for many of the most common skin infections. However, many...
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iPS Cell Differentiation01:22

iPS Cell Differentiation

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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Renewal of Skin Epidermal Stem Cells01:12

Renewal of Skin Epidermal Stem Cells

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The skin is divided into epidermis, dermis, and hypodermis, the skin's outermost, middle, and inner layers. The human epidermal layer regularly undergoes renewal, where old, dead cells are replaced by new cells. Epidermal stem cells or EpiSCs divide and differentiate to restore the lost cells. For the renewal process, some EpiSCs continuously self-renew. In contrast, few others differentiate into transit-amplifying cells, which later form prickle or spinous cells, followed by granular...
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Related Experiment Video

Updated: May 1, 2026

Development of an Economical DNA Delivery System by "Acufection" and its Application to Skin Research
09:42

Development of an Economical DNA Delivery System by "Acufection" and its Application to Skin Research

Published on: April 19, 2017

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Gene therapy for skin diseases.

Emily Gorell1, Ngon Nguyen, Alfred Lane

  • 1Department of Dermatology, Stanford School of Medicine, Palo Alto, California 94305.

Cold Spring Harbor Perspectives in Medicine
|April 3, 2014
PubMed
Summary

Gene therapy offers promising treatments for skin diseases by introducing genetic material to correct pathological processes. Further research is needed to address safety concerns for wider clinical application of this innovative gene therapy approach.

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Area of Science:

  • Dermatology
  • Molecular Biology
  • Biotechnology

Background:

  • The skin's unique properties make it a suitable target for gene therapy applications.
  • Gene therapy aims to correct genetic defects underlying various skin conditions.
  • Current research explores gene therapy for multiple cutaneous diseases.

Purpose of the Study:

  • To review the potential of gene therapy for treating skin diseases.
  • To discuss the mechanisms and methods of gene delivery to skin cells.
  • To identify challenges and future directions for clinical gene therapy in dermatology.

Main Methods:

  • Review of existing literature on gene therapy for cutaneous diseases.
  • Discussion of viral and nonviral vector systems for gene delivery.
  • Analysis of current advancements and limitations in the field.

Main Results:

  • Gene therapy has shown promising results in preclinical and some clinical studies for skin conditions.
  • Various viral vectors (e.g., lentivirus, adenovirus) and nonviral methods are employed for gene delivery.
  • Significant progress has been made in understanding gene expression modulation in skin cells.

Conclusions:

  • Gene therapy holds considerable potential for treating a range of skin disorders.
  • Addressing safety concerns and optimizing delivery methods are crucial for clinical translation.
  • Further research and development are necessary to overcome existing challenges and ensure the safe and effective use of gene therapy in dermatology.