Latest Advances in Targeted Molecular Therapies for Genetic Disease
A special issue of International Journal of Molecular Sciences (ISSN 1422-0067). This special issue belongs to the section "Molecular Pathology, Diagnostics, and Therapeutics".
Deadline for manuscript submissions: 31 July 2026 | Viewed by 644
Editor
Interests: prenatal; sequencing; breast cancer; ovarian cancer; homologous recombination deficiency; Romanian cohort; genetics; biomarkers; diagnosis; therapies; personalized medicine
Special Issue Information
Dear Colleagues,
This Special Issue aims to provide a comprehensive and critical overview of the latest advances in targeted molecular therapies for genetic diseases, highlighting their translational relevance and clinical impact. It brings together state-of-the-art research and authoritative reviews that explore innovative therapeutic strategies designed to address the underlying molecular and genetic mechanisms of inherited disorders. By directly addressing disease-causing molecular defects, these approaches represent a paradigm shift from symptomatic management toward mechanism-based intervention in genetic disorders.
The scope of the Issue encompasses both rare and common genetic diseases, highlighting how advances in molecular biology, genomics, and bioinformatics have accelerated the development of personalized and mechanism-driven treatments. Targeted molecular therapies seek to elucidate and therapeutically exploit the genetic and biochemical alterations underlying inherited diseases, with the overarching goal of developing more precise, effective, and durable treatment strategies.
Topics of interest include, but are not limited to, gene replacement and gene-editing strategies; RNA-based and antisense therapies; small-molecule and biologic agents targeting pathogenic molecular pathways; genomic and epigenomic profiling of genetic diseases; biomarkers for diagnosis, prognosis, and therapeutic efficacy; preclinical and clinical studies of precision therapies; and ethical, regulatory, and implementation challenges associated with advanced molecular interventions.
Dr. Viorica-Elena Rădoi
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-anonymized peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. International Journal of Molecular Sciences is an international peer-reviewed open access semimonthly journal published by MDPI.
Please visit the Instructions for Authors page before submitting a manuscript. There is an Article Processing Charge (APC) for publication in this open access journal. For details about the APC please see here. 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
- genetic diseases
- precision medicine
- gene editing
- RNA-based therapeutics
- genomic profiling
- translational research
Benefits of Publishing in a Special Issue
- Ease of navigation: Grouping papers by topic helps scholars navigate broad scope journals more efficiently.
- Greater discoverability: Special Issues support the reach and impact of scientific research. Articles in Special Issues are more discoverable and cited more frequently.
- Expansion of research network: Special Issues facilitate connections among authors, fostering scientific collaborations.
- External promotion: Articles in Special Issues are often promoted through the journal's social media, increasing their visibility.
- Reprint: MDPI Books provides the opportunity to republish successful Special Issues in book format, both online and in print.
Further information on MDPI's Special Issue policies can be found here.
