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Biomedicines
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State-of-the-Art Molecular and Translational Medicine in USA
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State-of-the-Art Molecular and Translational Medicine in USA

A special issue of Biomedicines (ISSN 2227-9059). This special issue belongs to the section "Molecular and Translational Medicine".

Deadline for manuscript submissions: 31 December 2025 | Viewed by 784

Special Issue Editors

Dr. Maaike van Gerwen

E-Mail Website
Guest Editor
Department of Otolaryngology-Head and Neck Surgery, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
Interests: environmental pollutants; endocrine disrupting chemicals; cancer
Special Issues, Collections and Topics in MDPI journals
Dr. Stephanie J. Tuminello

E-Mail Website
Guest Editor
Thoracic Surgery and Institute for Translational Epidemiology, Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA
Interests: immunotherapy; genetics; personalized medicine; cancer epidemiology; molecular epidemiology; translational research
Special Issues, Collections and Topics in MDPI journals

Special Issue Information

Dear Colleagues,

Molecular and translational research represents a multidisciplinary field at the intersection of basic science and clinical application. It focuses on understanding biological processes at the molecular level and translating these insights into practical medical advancements, including diagnostics, therapeutics, and preventative strategies.

Key areas of molecular research include genomics, proteomics, metabolomics, and cellular biology, which aim to unravel the mechanisms underlying diseases. Translational research builds on this foundation, emphasizing the "bench-to-bedside" approach—bridging laboratory discoveries with clinical implementation. This process involves identifying molecular targets, developing experimental therapies, and validating these interventions in preclinical and clinical studies.

This field plays a pivotal role in precision medicine, which involves tailoring treatments based on individual genetic and molecular profiles. Innovations such as gene editing, biomarker discovery, and immunotherapies are direct outcomes of this research. Collaboration among biologists, clinicians, bioinformaticians, and pharmaceutical industries is essential for advancing this field and improving patient outcomes.

Molecular and translational research continues to evolve, driven by advancements in technology such as CRISPR, next-generation sequencing, and artificial intelligence, promising new frontiers in disease management and healthcare innovation.

For this Special Issue, we are seeking submissions of original research articles or reviews presenting impactful advances in the field of molecular and translational medicine in the USA.

Dr. Maaike van Gerwen
Dr. Stephanie J. Tuminello
Guest Editors

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 100 words) can be sent to the Editorial Office for announcement on this website.

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. Biomedicines is an international peer-reviewed open access monthly 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 2600 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

  • diagnostics
  • therapeutics
  • prevention
  • cellular biology
  • disease mechanisms
  • precision medicine
  • biomarker discovery
  • immunotherapies
  • bioinformatics

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Published Papers (1 paper)

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Review

27 pages, 1466 KiB  
Review
Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context
by Ayrton Bangolo, Behzad Amoozgar, Lili Zhang, Sarvarinder Gill, Daniel Lushimba Milolo, Justin Ngindu Kankonde, Claude Mbuyi Batakamuna, Robert Tassan, Christina Cho, John Bukasa-Kakamba and Kelley Mowatt-Pesce
Biomedicines 2025, 13(8), 2022; https://doi.org/10.3390/biomedicines13082022 - 19 Aug 2025
Viewed by 376
Abstract
Hemophilias and hemoglobinopathies—including hemophilias A and B, sickle cell disease (SCD), and β-thalassemia—are debilitating genetic disorders associated with significant global health burdens. While traditional management has centered on factor replacement and transfusions, these approaches remain palliative, with limited access and durability in many [...] Read more.
Hemophilias and hemoglobinopathies—including hemophilias A and B, sickle cell disease (SCD), and β-thalassemia—are debilitating genetic disorders associated with significant global health burdens. While traditional management has centered on factor replacement and transfusions, these approaches remain palliative, with limited access and durability in many regions. Recent advances in immune-based therapeutics (e.g., emicizumab, concizumab, crizanlizumab), viral vector-mediated gene addition (e.g., Roctavian, Hemgenix), and gene-modified autologous stem cell therapies (e.g., Zynteglo, Casgevy) have ushered in a new era of disease-modifying and potentially curative interventions. These therapies offer durable efficacy and improved quality of life, particularly in adult populations. However, implementation remains uneven across global health systems due to high costs, limited infrastructure, and regulatory heterogeneity. Additionally, ethical considerations such as long-term surveillance, informed consent in vulnerable populations, and social perceptions of genetic modification present ongoing challenges. Innovations such as multiplex genome editing, immune-evasive donor platforms, synthetic biology, and AI-driven treatment modeling are poised to expand therapeutic horizons. Equitable access, particularly in regions bearing the highest disease burden, will require collaborative funding strategies, regional capacity building, and inclusive regulatory frameworks. This review summarizes the current landscape of curative therapy, outlines implementation barriers, and calls for coordinated international action to ensure that transformative care reaches all affected individuals worldwide. Full article
(This article belongs to the Special Issue State-of-the-Art Molecular and Translational Medicine in USA)
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