Review
Genetics & Heredity
Jennifer Lim, Venessa Chin, Kirsten Fairfax, Catia Moutinho, Dan Suan, Hanlee Ji, Joseph E. Powell
Summary: Genomics has been widely used in clinical practice, leading to innovations in genetic screening, rare disease diagnosis, and molecularly guided therapy choice. Single-cell technologies have the potential to revolutionize genomic medicine by unveiling the cellular heterogeneity underlying disease pathology and mechanisms. However, challenges in translating single-cell genomics from research to clinical practice need to be addressed.
NATURE REVIEWS GENETICS
(2023)
Article
Chemistry, Multidisciplinary
Annette I. S. van den Berg, Chae-Ok Yun, Raymond M. Schiffelers, Wim E. Hennink
Summary: This review discusses the clinical applications of gene therapy using nucleic acids and the development of carrier systems, highlighting the drawbacks of viral vectors and the potential of non-viral carrier systems. It focuses on polymer-based nucleic acid formulations, known as polyplexes, summarizing different polymeric systems and addressing cellular barriers encountered by polyplexes. Attention is given to the clinical status of non-viral nucleic acid formulations.
JOURNAL OF CONTROLLED RELEASE
(2021)
Review
Biochemistry & Molecular Biology
Divya Sharma, Sanjay Arora, Jagdish Singh, Buddhadev Layek
Summary: Gene therapy aims to transfer genetic materials into targeted cells for disease treatment or prevention. Nonviral vectors have gained attention for their safety, payload capacity, and stealth abilities, but face challenges in overcoming intracellular and extracellular barriers. This review highlights current advancements in nonviral gene delivery systems and their future potential.
INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES
(2021)
Review
Pharmacology & Pharmacy
Gink N. Yang, Philippe Ke Roberts, Jesse Gardner-Russell, Manisha H. Shah, Terry A. Couper, Zhuoting Zhu, Graeme A. Pollock, Gregory J. Dusting, Mark Daniell
Summary: Corneal diseases, particularly corneal scarring, are a major cause of visual impairment and blindness worldwide. They primarily affect younger individuals and disproportionately impact those in poorer and remote areas. Traditional medical strategies are ineffective in preventing scarring, and corneal transplantation faces numerous challenges. However, emerging therapeutic options such as gene therapy, protein therapy, and cell therapy offer promising solutions. This article summarizes recent preclinical and clinical studies on these emerging therapies, highlighting their potential to prevent the development of corneal scarring. (c) 2023 Published by Elsevier Inc.
PHARMACOLOGY & THERAPEUTICS
(2023)
Review
Biotechnology & Applied Microbiology
Zongmin Zhao, Aaron C. Anselmo, Samir Mitragotri
Summary: Gene therapies using viral vectors have shown promising outcomes in treating various diseases that were once considered incurable. The use of viral vectors allows for efficient delivery of gene therapeutics into target cells, leading to the approval of multiple products and ongoing clinical trials exploring their therapeutic potential.
BIOENGINEERING & TRANSLATIONAL MEDICINE
(2022)
Review
Biotechnology & Applied Microbiology
Alexander Curreri, Disha Sankholkar, Samir Mitragotri, Zongmin Zhao
Summary: RNA therapeutics are actively studied for their potential as a therapeutic modality in both preclinical and clinical studies. With the ongoing COVID-19 pandemic, they have gained significant attention and are widely discussed. Since their first approval in 1998, research on RNA therapeutics has advanced to explore new therapeutic targets and delivery strategies to improve their safety and effectiveness. This review provides an overview of currently approved RNA therapeutics, their mechanisms of action, and strategies for enhancing their clinical use. It also analyzes ongoing clinical trials and discusses the challenges and potential strategies for clinical translation.
BIOENGINEERING & TRANSLATIONAL MEDICINE
(2023)
Review
Pharmacology & Pharmacy
Yeri Alice Rim, Yoojun Nam, Narae Park, Ji Hyeon Ju
Summary: Minicircles, as non-viral vectors, have shown promise in delivering genes efficiently and safely into cells, particularly in studies related to cartilage tissues and diseases like osteoarthritis and rheumatoid arthritis.
Article
Engineering, Biomedical
Nan Hu, Li Zhu, Li Zhang, Jing Wang, Yanhua Wang, Jing Luo, Lan He, Zhiming Hao, Long Zhang
Summary: Oral delivery of TNF-alpha shRNA mediated by non-pathogenic yeast can effectively inhibit the inflammatory response in rheumatoid arthritis without toxic effects on peripheral blood and major organs.
MATERIALS TODAY BIO
(2022)
Article
Immunology
Anuja Singh, Tapan Behl, Aayush Sehgal, Sukhbir Singh, Neelam Sharma, Tanveer Naved, Saurabh Bhatia, Ahmed Al-Harrasi, Prasun Chakrabarti, Lotfi Aleya, Celia Vargas-De-La-Cruz, Simona Bungau
Summary: Rheumatoid arthritis is an autoimmune disease characterized by severe inflammation that damages tendons, cartilage, and bones. B cells play a significant role in the pathogenesis of RA, and targeting B cells has shown therapeutic efficacy in treating autoantibody-related conditions, including RA. The development of B-cell-targeted therapies has led to a better understanding of the diverse roles of B cells in autoimmune diseases and their potential as effective treatment targets.
INTERNATIONAL IMMUNOPHARMACOLOGY
(2021)
News Item
Biotechnology & Applied Microbiology
Megan Cully
Summary: Exosomes, small extracellular vesicles shed by cells, have long been considered promising drug delivery systems for various biologic payloads, with the first ones now in clinical trials.
NATURE REVIEWS DRUG DISCOVERY
(2021)
Review
Nanoscience & Nanotechnology
Kave Moloudi, Ali Khani, Masoud Najafi, Rasool Azmoonfar, Mehdi Azizi, Houra Nekounam, Mahsa Sobhani, Sophie Laurent, Hadi Samadian
Summary: Radiotherapy is an important treatment for cancer, but it can affect normal cells and some tumor cells may be resistant to it. Gold nanoparticles have shown potential as radiosensitizers, but they have not been approved for clinical use yet. The effectiveness of gold nanoparticles depends on their concentration and the energy deposition during radiotherapy. This review discusses the challenges and biases in using gold nanoparticles as radiosensitizers, as well as suggests solutions and areas for future research.
WILEY INTERDISCIPLINARY REVIEWS-NANOMEDICINE AND NANOBIOTECHNOLOGY
(2023)
Review
Immunology
Matteo Vecellio, Vivien Xanath Hake, Connor Davidson, Maria Cristina Carena, B. Paul Wordsworth, Carlo Selmi
Summary: Psoriatic arthritis (PsA) is a chronic inflammatory disease associated with psoriasis, with genetic predisposition playing a key role in its development. The IL-23/IL-17 axis is highlighted as important in the immunopathogenesis of PsA.
FRONTIERS IN IMMUNOLOGY
(2021)
Review
Engineering, Biomedical
Sabrina N. Campelo, Po-Hsun Huang, Cullen R. Buie, Rafael V. Davalos
Summary: In the past decade, electroporation-based technologies have gained popularity in various fields including molecular biology, food sanitation, and therapeutic applications. This article provides an overview of the biophysics behind electroporation and discusses the advancements in single-cell and in vivo electroporation technologies.
ANNUAL REVIEW OF BIOMEDICAL ENGINEERING
(2023)
Article
Biochemistry & Molecular Biology
Aleksandra Petrovic Fabijan, Jonathan Iredell, Katarzyna Danis-Wlodarczyk, Razieh Kebriaei, Stephen T. T. Abedon
Summary: Phage therapy is a medical form of biological control using phages as antibacterial agents to combat bacterial infections. It is gaining renewed interest due to its potential in providing safe and effective cures for infections resistant to traditional antibiotics. However, further implementation and mainstream acceptance of phage therapy face challenges in biology, regulations, and economics.
Review
Plant Sciences
Mariane S. Sousa-Baena, Jose Hernandes-Lopes, Marie-Anne Van Sluys
Summary: Climbing plants use voluble organs such as tendrils and modified stems to climb neighboring plants by growing towards shaded areas and responding to mechanical stimuli. The molecular pathways underlying the growth and development of these organs are largely unknown, with epigenetics and sensory biology being highlighted as emerging frontiers in research on climbing plants. Recent advancements in the study of plants' mechanosensory system are discussed in the context of climbing habits.
CURRENT OPINION IN PLANT BIOLOGY
(2021)