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7.2
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Biologics
Special Issues
Gene and Stem Cell Therapies for Inherited Metabolic Disorders
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Gene and Stem Cell Therapies for Inherited Metabolic Disorders

A special issue of Biologics (ISSN 2673-8449).

Deadline for manuscript submissions: 25 September 2026 | Viewed by 1181

Special Issue Editor

Prof. Dr. Paul Gissen

E-Mail Website
Guest Editor
Institute for Health Research, Great Ormond Street Hospital Biomedical Research Centre, University College London, London WC1N 1EH, UK
Interests: DNA and RNA therapy; biochemistry and cell biology; genetics; lysosomal storage disorders; paediatric metabolic medicine; rare paediatric diseases, use of viral and non-viral vector for gene therapy delivery
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Inherited metabolic disorders (IMDs), including lysosomal storage disorders and a broad spectrum of inborn errors of metabolism, represent some of the most severe and life-limiting rare paediatric diseases. Recent advances across genetics, biochemistry, and cell biology have transformed our understanding of disease mechanisms and revealed new opportunities for targeted intervention. In parallel, major progress in DNA- and RNA-based therapeutic technologies—from gene replacement and editing to RNA modulation and antisense approaches—has accelerated the development of precision therapies capable of addressing the underlying molecular defects.

This Special Issue highlights emerging innovations in gene and stem cell therapies designed to correct metabolic dysfunction at its source. We welcome studies spanning viral and non-viral delivery platforms, including AAV, lentiviral, and genome-editing vectors, as well as nanoparticle-, lipid-, and polymer-based systems. Contributions exploring engineered cell therapies, including haematopoietic stem cell gene therapy and ex vivo genome editing, are equally encouraged.

By bringing together cutting-edge research across discovery science, translational studies, and early-phase clinical development, this Special Issue aims to showcase the rapidly expanding therapeutic landscape for IMDs and to catalyse further progress toward safe, durable, and transformative treatments for affected children and their families.

Prof. Dr. Paul Gissen
Guest Editor

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All submissions that pass pre-check are peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 250 words) can be sent to the Editorial Office for assessment.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Biologics is an international peer-reviewed open access quarterly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1200 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.

Keywords

  • DNA and RNA therapy
  • inborn errors of metabolism
  • individualised therapies
  • lysosomal storage disorders
  • paediatric metabolic medicine
  • rare inherited diseases
  • use of viral and non-viral vector for gene therapy delivery

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Published Papers (1 paper)

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38 pages, 712 KB  
Review
The Evolving Landscape of Fetal Therapy: Surgical Interventions and Emerging Biologics
by Berna Seker-Yilmaz, Melissa Hill, Giovanni Baranello, Stavros Loukogeorgakis, Paolo De Coppi, Paul Gissen and Lyn S. Chitty
Biologics 2026, 6(2), 11; https://doi.org/10.3390/biologics6020011 - 13 Apr 2026
Viewed by 801
Abstract
Fetal therapy has evolved into a rapidly advancing field with the potential to alter the natural history of many severe congenital and genetic disorders before irreversible injury occurs. Progress in prenatal imaging, molecular diagnostics, and fetal intervention techniques now enables the earlier identification [...] Read more.
Fetal therapy has evolved into a rapidly advancing field with the potential to alter the natural history of many severe congenital and genetic disorders before irreversible injury occurs. Progress in prenatal imaging, molecular diagnostics, and fetal intervention techniques now enables the earlier identification of disease and, in select settings, targeted prenatal treatment. This review synthesizes the current landscape of fetal therapies, spanning established surgical interventions for structural anomalies and emerging biologic and molecular approaches, including enzyme replacement therapy, stem cell-based strategies, gene therapy, and gene editing. The intrauterine environment provides a distinct therapeutic context, with developmental plasticity, immune immaturity, enhanced tissue accessibility, and relatively permissive central nervous system exposure that together define a time-sensitive window for intervention. Preclinical studies and early clinical experience across both structural anomalies and genetic disorders, including lysosomal storage disorders, osteogenesis imperfecta, and spinal muscular atrophy, support the premise that prenatal treatment can preserve organ development and improve pediatric outcomes. However, translation remains constrained by procedural risks, uncertainty regarding long-term safety and durability, ethical and regulatory complexities, and challenges with equitable access, alongside the need for robust comparative evidence versus early postnatal therapy. As the field advances, multidisciplinary collaboration, rigorous trial design with meaningful developmental endpoints, and ethically grounded implementation frameworks will be essential to guide responsible clinical adoption and maximize benefit for children and families. Full article
(This article belongs to the Special Issue Gene and Stem Cell Therapies for Inherited Metabolic Disorders)
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