Gene and Stem Cell Therapies for Inherited Metabolic Disorders

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.