Tremendous developments in biology and medicine have been achieved during the past few decades. However, it takes far too long for these developments to become available outside of the laboratory. It is an extremely important task to translate these great scientific achievements into everyday practice.
However, the science alone is not enough which is why we keep an eye on adapting cutting edge technologies and envisioning the future. Here are some research opportunities we think will be crucial for future biotechnology and medical advancements.
Gene therapy enables the efficient and safe delivery of genes into a patient’s cells. It has a long history having been practiced first in 1980. However, early clinical failures, the death of a single patient due to a severe immune reaction and the observation of cancer as a side-effect, set back the trials and horrified the public, halting research for decades.
Since 2006 though, gene therapy has been gaining new attention. Safety has improved enabling new investments by venture capital and big pharmaceutical firms. This is shown by the 3.5x increase in the number of gene therapy companies over the last four years (69 to 255; ARM data).
Oncology and rare diseases dominate the field with other disease comprising only about one third of cases. Viral delivery is used as the insertion vehicle in two thirds of cases and within these recombinant Adeno-associated viruses (rAAVs) are followed by Lentivirus, Adenovirus, Retrovirus and Vaccinia virus.
Although rAAVs are less immunogenic than other viruses, they can trigger different layers of the host immune system. A major difficulty is finding the effective but non-immunogenic dose. Most patients have previously had AAV infections and so have developed an immune response to AAV particles. Another disadvantage is the cargo size being limited to at most 5 kb in AAV vectors. AAVs have single stranded DNA genomes, so need to be converted to dsDNS first before integration can happen; during second strand synthesis, errors can form leading to inefficient transgene expression and other problems. That viral DNA preferentially integrates into active genes can be a major disadvantage itself. Additionally, integration happens through non-homologous end-joining which frequently is associated with chromosomal rearrangement and deletions of large sections of DNA.
The most popular vector has fundamental weaknesses, but other viral delivery vehicles share similar difficulties: immunogenicity and cytotoxicity, limited cargo size and insertional mutagenesis. It is obvious that current delivery vehicles are far from ideal and there is still great room for improvement or for completely novel approaches. There is great demand for highly efficient and safe gene delivery.
3D printing is a revolutionary technology which has already appeared in medicine and biology. There are four core uses of 3D printing associated with recent innovations in the medical field: creating tissues and organoids, surgical tools, patient-specific surgical models and custom-made prosthetics. Some interesting current results:
For tissue building, a scaffold is built and then cells placed in specific locations on this scaffold. 3D printing can be used for both. Bio-printing enables precise placement of high-density cells in desired locations, enabling native tissues to be mimicked by arranging multiple types of cells into an ordered structure. 3D bio-printing has, for example, been used to product cartilage tissue by printing chondrocytes onto hydrogel scaffolds. Current research is also focused on developing skin to help burn victims.
It is not possible to completely prevent the transfer of infectious agents such as MRSA between patients via multi use surgical tools. But these traditional tools are too expensive to be used only once. 3D printed tools could be cheap enough to be single use only and could also be completely recyclable.
In our opinion, this is the beginning of a profound change that will shape the future of medicine. We will use complex nano-tools and nano-machines for medical purposes, custom lab-on-a-chip devices for diagnostics, intelligent surgical devices and bio-printed tissues and organs. There is a great need for bio-compatible, biodegradable non-immunogenic materials, smart composites and higher printing resolutions.