Review
Medicine, General & Internal
Giacomo Frati, Annarita Miccio
Summary: Beta-hemoglobinopathies are genetic disorders caused by mutations affecting adult hemoglobin production or structure, leading to anemia and multi-organ damage. Current therapeutic options include symptomatic care, blood transfusions, and pharmacological treatments, with lentiviral-mediated gene therapy showing promise but being costly and variable in outcomes. Genome editing has emerged as a valuable tool for developing curative strategies for beta-hemoglobinopathies, with a focus on correcting defective beta-globin genes or inducing fetal hemoglobin production.
JOURNAL OF CLINICAL MEDICINE
(2021)
Review
Biotechnology & Applied Microbiology
Isabella Hetherington, Hana Totary-Jain
Summary: Atherosclerosis is the primary pathology underlying many cardiovascular diseases and is a major global cause of death. Current therapies focus on controlling risk factors rather than targeting the cells that cause plaque formation. This review summarizes the milestones, strengths, and limitations of current anti-atherosclerotic therapies while also exploring potential game-changing technologies such as nanomedicine, mRNA therapeutics, and gene editing for precision medicine in CVD clinical practice.
Review
Biochemistry & Molecular Biology
Bryce Wallis, Katherine Redd Bowman, Phong Lu, Carol S. Lim
Summary: It is well known that p53 mutations are common in cancer tumors, including ovarian cancer. Ovarian cancer is often diagnosed at later stages and has a low survival rate, especially for high-grade serous ovarian carcinoma (HGSOC) which has a high prevalence of p53 mutations. Previous p53-based therapies for ovarian cancer have faced challenges and limited success. This review discusses the failures of historical p53-targeted therapeutics and the potential for new generation therapies.
Review
Pharmacology & Pharmacy
Catia Domingues, Ivana Jarak, Francisco Veiga, Marilia Dourado, Ana Figueiras
Summary: The paradigm of pediatric drug development is evolving to address population-specific issues. The off-label use of adult medicines for pediatric patients persists, compromising proper evaluation. Nanotechnology-based delivery systems and advanced therapy medicinal products (ATMPs) offer potential solutions. The involvement of a multidisciplinary workforce is crucial for standardized guidelines and quick translation.
Review
Biochemistry & Molecular Biology
Mohamed Sabra, Catherine Karbasiafshar, Ahmed Aboulgheit, Sidharth Raj, M. Ruhul Abid, Frank W. Sellke
Summary: Cardiovascular diseases remain the leading cause of death globally, with new therapeutic approaches showing limited success in clinical practice. Challenges in patient selection, endpoint measurements, comorbidities, and delivery systems persist, but the field of therapeutic angiogenesis is evolving with novel strategies and bioengineering approaches emerging.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2021)
Article
Biotechnology & Applied Microbiology
Evangelia Yannaki, Nikoletta Psatha, Anastasia Papadopoulou, Takis Athanasopoulos, Achilleas Gravanis, Maria G. Roubelakis, Panagiotis Tsirigotis, Achilles Anagnostopoulos, Nicholas P. Anagnou, George Vassilopoulos
Summary: Gene therapy, a relatively new field with around four decades of development, has entered the clinical stage with a focus on hemoglobinopathies and hematopoietic stem cells. Research efforts have been made to efficiently implement gene therapy through advancements in HSC biology and gene expression technologies, potentially transforming the therapeutic landscape for genetic diseases in the near future.
HUMAN GENE THERAPY
(2021)
Editorial Material
Hematology
Ariel Koren
Summary: This article discusses different approaches to treating beta-thalassemia patients. The report highlights significant differences in facilities and economic resources available for patient care, which is a major concern. Managing thalassemia should be a global healthcare priority, which may involve establishing national and international registries, as well as implementing national programs for screening at-risk couples and providing preventive measures to prevent the birth of thalassemia patients.
BRITISH JOURNAL OF HAEMATOLOGY
(2023)
Article
Medicine, General & Internal
Zhijun Zhou, Barish H. Edil, Min Li
Summary: Gastrointestinal cancers, especially pancreatic cancer, pose a significant challenge to public health. Neoadjuvant chemotherapy offers some patients the chance for radical resection. An optimal chemotherapy regimen should maximize anti-tumor efficacy while maintaining manageable safety. Surgical advancements further enhance outcomes for these patients.
Review
Biotechnology & Applied Microbiology
Xiao Hu, Yu-Sen Zhang, Yi-Chao Liu, Na Wang, Xian-Tao Zeng, Ling-Ling Zhang
Summary: Photodynamic therapy (PDT) and sonodynamic therapy (SDT) have been developed to treat solid tumors, and they are particularly suitable for urological tumors. With the advancement of nanobiotechnology, new photo/sonosensitizers have significant advantages over traditional ones, offering great potential for cancer treatment. Clinical trials and multidisciplinary participation are needed for the clinical translation of these promising photo/sonosensitizers.
JOURNAL OF NANOBIOTECHNOLOGY
(2022)
Review
Biochemistry & Molecular Biology
Han Yang, Yufang Liu, Longqing Chen, Juanjuan Zhao, Mengmeng Guo, Xu Zhao, Zhenke Wen, Zhixu He, Chao Chen, Lin Xu
Summary: Lung cancer is a prevalent and deadly disease, with limited treatment options, especially for advanced stage patients. However, recent advances in miRNA-based therapies show potential in improving prognosis and overcoming drug resistance. Clinical trials have demonstrated promising outcomes for siRNA and miRNA-based therapies. This review discusses the current state of miRNA-based therapies in lung cancer treatment and their challenges, offering new ideas for the development of novel lung cancer therapies.
Review
Pharmacology & Pharmacy
Marie-Anne Morren, Eric Legius, Fabienne Giuliano, Smail Hadj-Rabia, Daniel Hohl, Christine Bodemer
Summary: Genodermatoses are rare inherited skin diseases that often affect other organs, causing significant impacts on patients' health and well-being. Advances in molecular genetics and translational research have provided opportunities for innovative treatments, including intervention in cell-signaling pathways, suppression of inflammation, and correction of genetic defects. These technologies vary in terms of personalized, short-term, and permanent therapeutic effects. Prevention of transmission to future generations is also an important goal for severe genodermatoses.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Chemistry, Medicinal
Michaela Sharpe, Jacqueline Barry, Panos Kefalas
Summary: With the maturing of the cell and gene therapy field, advancements in manufacturing, logistics, regulation, reimbursement, and healthcare facilities are necessary to support the wider clinical adoption of promising treatments. Collaboration among stakeholders is crucial to develop the required processes, skilled workforce, and infrastructure needed for the transition of more treatments from clinical trials to marketed products.
JOURNAL OF PHARMACEUTICAL SCIENCES
(2021)
Review
Biochemistry & Molecular Biology
Imogen R. Brooks, Adam Sheriff, Declan Moran, Jingbo Wang, Joanna Jackow
Summary: Genodermatoses, a group of inherited skin diseases, pose significant challenges due to their severity and limited treatment options. This review explores the emerging field of gene editing for genodermatoses, specifically discussing tools such as CRISPR-Cas9, homology-directed repair, base editing, and prime editing for correcting pathogenic mutations. The optimization of editing efficiency, minimization of off-target edits, and delivery tools are crucial for clinical translation. The aim of this review is to provide important considerations for researchers developing gene editing approaches for genodermatoses.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Review
Chemistry, Medicinal
Atif Zafar, Wei Wang, Gang Liu, Xinjie Wang, Wa Xian, Frank McKeon, Jennifer Foster, Jia Zhou, Ruiwen Zhang
Summary: Neuroblastoma, the most common pediatric solid tumor, exhibits genetic, morphological, and clinical heterogeneity, making current treatment modalities less effective. Understanding the molecular signatures and genetic variations in neuroblastoma pathogenesis is crucial for developing safer and more effective treatments. Advanced omics techniques have identified genetic alterations and dysfunctional pathways driving neuroblastoma, paving the way for new targeted therapies to improve patient outcomes.
MEDICINAL RESEARCH REVIEWS
(2021)
Review
Pharmacology & Pharmacy
Laura Grech, Karen Borg, Joseph Borg
Summary: Beta-thalassaemia, a significant haemoglobinopathy, is currently managed with treatments such as repeated transfusions and iron-chelating therapy, while novel approaches like gene therapy and gene editing are being developed to address the underlying causes of the disease. These new therapies aim to correct the imbalance of globin chains, treat ineffective erythropoiesis, and improve iron overload associated with beta-thalassaemia.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2022)