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Hair Growth and Disorders / Genetic and Rare Skin Diseases. · Journal article
Frontiers in Bioengineering and Biotechnology · August 24, 2026
A consensus or society position rather than new primary data.
This is a narrative review examining the translational state of gene therapy for inherited genodermatoses, particularly epidermolysis bullosa and ichthyoses. The review documents proof-of-concept achievements in ex vivo stem cell therapy and emerging genome editing platforms (CRISPR, base editors, prime editors), while identifying substantial barriers—including epidermal stem cell targeting, skin barrier penetration, off-target control, manufacturing scalability, and long-term safety—that presently limit broad clinical implementation.
Narrative review. Inherited genodermatoses, particularly epidermolysis bullosa and ichthyoses, and the current and emerging therapeutic approaches for these rare monogenic skin disorders..
Ex vivo gene therapy has demonstrated proof-of-concept for long-term tissue regeneration in epidermolysis bullosa skin patients using genetically corrected epidermal stem cells. Programmable genome editing platforms including CRISPR/Cas nucleases, base editors and prime editors enable mutation-specific correction strategies for both recessive and dominant genodermatoses. In vivo topical approaches are emerging to enable direct targeting of skin, expanding therapeutic options beyond ex vivo strategies.
No quantitative data on efficacy, safety, or durability from clinical trials are provided. Comparative effectiveness or adverse event profiles are not reported.
Clinicians and researchers should recognize that while gene therapy has achieved proof-of-concept in epidermolysis bullosa through ex vivo stem cell approaches and multiple genome editing platforms are advancing, substantial translational barriers remain before these therapies become broadly clinically accessible. Current management remains palliative, and disease-modifying therapies remain investigational.
A comprehensive review synthesizing current state, clinical milestones, and translational barriers in gene therapy for genodermatoses, offering expert perspective on therapeutic landscape and implementation challenges.
As stated by the source record.
Quoted from the source exactly as published.
Clinicians and researchers should recognize that while gene therapy has achieved proof-of-concept in epidermolysis bullosa through ex vivo stem cell approaches and multiple genome editing platforms are advancing, substantial translational barriers remain before these therapies become broadly clinically accessible. Current management remains palliative, and disease-modifying therapies remain investigational.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
Inherited genodermatoses are a heterogeneous group of rare monogenic disorders. Among these, epidermolysis bullosa (EB) and ichthyoses represent paradigmatic disorders characterized by severe skin fragility and hyperkeratosis, respectively, and impaired barrier function, often with profound effects on quality of life and systemic health. Current management remains largely palliative, underscoring the urgent need for disease-modifying therapies. Over the past 2 decades, advances in epithelial stem cell biology, vector engineering and genome editing technologies have transformed the therapeutic landscape for genodermatoses. Ex vivo gene therapy has provided the first proof that genetically corrected epidermal stem cells can achieve long-term tissue regeneration in EB skin patients, establishing a new paradigm for regenerative medicine. In parallel, the emergence of programmable genome engineering platforms, including CRISPR/Cas nucleases, base editors and prime editors, have enabled increasingly precise strategies for mutation-specific correction in both recessive and dominant disorders. Furthermore, the development of in vivo topical approaches is expanding the possibility of directly targeting the skin. Despite these advances, substantial translational barriers continue to limit broad clinical implementation. Efficient and durable targeting of epidermal stem cells within a highly regenerative tissue, together with safe delivery across the skin barrier, stringent control of off-target activity, scalable manufacturing and demonstration of long-term safety, remain major challenges for the clinical translation of these approaches. In this Review, we discuss the current state of gene therapy for genodermatoses, highlighting key clinical milestones, emerging genome editing technologies and next-generation delivery systems. We further examine the biological and regulatory challenges that need to be overcome to bridge the gap between experimental innovation and clinically accessible therapies for patients with inherited skin diseases.
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