Gene therapy is a promising therapeutic approach that has experienced significant growth in recent decades, with gene nanomedicines reaching the clinics. However, translating more gene nanomedicines from bench to bedside is still challenging. The use of robust preclinical models that can better predict the [...]
Rare diseases collectively affect approximately 400 million patients worldwide [1]. Nevertheless, this number is expected to increase thanks to the rapid evolution of sequencing technologies that have identified more than 6,000 to 10,000 rare diseases [2]. However, most rare diseases still lack specific treatments, [...]
Exosomes, subcellular structures that act as specific carriers for transferring genetic material and information between cells, demonstrate the potential to become significant players in targeted drug delivery and personalized medicine. Their tumor-homing properties and ability to cross biological barriers are among the factors contributing [...]
Despite significant advancements in molecular medicine, obstacles such as low solubility and poor tissue accessibility impede the clinical translation of promising molecules and therapeutic agents. In the ever-evolving landscape of scientific research, nanotechnology emerges as a beacon of innovation, offering unique versatility to overcome [...]
The potential of non-viral lipid delivery systems for improving global health has been proven once again. From rare diseases to cancer, gene editing and gene therapies will undoubtedly benefit from the latest advances in nanomedicine. In February 2024, the results from a promising combined phase [...]
Melanoma, a type of skin cancer originating from melanocytes, has become a global health concern due to its rising incidence. It ranks as the fifth most prevalent cancer in men and the sixth most common in women [1]. To advance in the fight against this [...]
2024 is here full of new resolutions and challenges. And so, in an ever-evolving field such as medicine, it's inevitable to wonder what exciting advances the new year will bring. Nature medicine has asked leading researchers to discover what clinical trials may impact medicine in [...]
The versatility of nanotechnology to fight different diseases in diverse therapeutic areas is unprecedented. Combining nanoparticles and mRNA to treat autoimmune diseases could be the latest blockbuster, adding the development of COVID-19 vaccines and the new announcement of promising results for a personalized mRNA vaccine [...]
Is it possible for a drug to go through a clinical trial more than once? Potent drugs with high therapeutic efficacy, such as the anticancer drug doxorubicin, generate adverse effects that reduce their application in clinical practice, limiting healthcare professionals' and patients' choices. Nanotechnology holds [...]
The Food and Drug Administration (FDA) has joined the UK medicines regulator and has approved the first CRISPR-Cas9 gene-editing therapy to treat sickle-cell disease [1]. UK expands approval to β-thalassemia. The approval of Casgevy, the therapy developed by Vertex and CRISPR Therapeutics, is the first [...]
