The Biggest Innovations in Biosciences

We are in the era of innovation and cutting-edge technology in biosciences and health. This article goes through some of the most remarkable technologies slowly conquering the world of biosciences.

Gene editing and CRISPR-Cas9

Gene editing is based on the idea that correcting the genetic mistake that causes a disease offers a permanent result than curing the symptoms. This technique allows scientists to alter the DNA of cells by deleting, adding or modifying genes. There are numerous ways to edit a gene.

The most widely used and revolutionary method for gene editing is CRISPR-Cas9, which

stands for Clustered Regularly Interspaced Short Palindromic Repeats and CRISPR-

associated protein 9. The process begins with the design of a synthetic RNA molecule,

known as guide RNA (gRNA) that matches the target gene sequence. The gRNA, combined with the Cas9 protein, forms a complex that is then introduced into the target cells. Cas9 acts like scissors, guided by the gRNA, to locate the precise location on the DNA where the genetic modification is intended. Once the target site is identified, Cas9 induces a break in the DNA strand. The cell's natural DNA repair mechanisms then come into play. The non- homologous end joining pathway introduces insertions and deletions at the site, resulting in gene knockout or inactivation. On the other hand, once a DNA template with homology to the sequences is present, the homology-directed repair pathway allows the incorporation of a desired genetic sequence, facilitating gene insertion or replacement.

Several other gene-editing techniques have been developed, each with unique approaches. Zinc Finger Nucleases (ZFNs) and Transcription Activator-Like Effector Nucleases (TALENs) are two examples. These methods also use proteins that act as molecular scissors to cut the DNA at specific locations. ZFNs use zinc finger proteins to bind to target DNA sequences, while TALENs use transcription activator-like effector proteins. As the field of gene editing rapidly advances, these diverse methods contribute to the expanding toolkit available for researchers and hold promise for addressing a wide array of applications, from medical treatments to agricultural improvements.

CAR T-cells

Chimeric antigen receptor T-cells (CAR T-cells) are a new type of immunotherapy,

considered to be the new fighters in the war on cancer. In general, immunotherapies use the patient’s immune system to fight the cancer. This therapy promises more specificity than traditional therapies and more permanent results. T-cells naturally exist in the human

organism, supporting the adaptive immune system. They are a group of lymphocytes in the blood or lymph tissue that target or kill specific pathogens. Each type of T-cell recognises specific pathogens. T-cells have proteins on their outer surface, called receptors and these receptors recognize specific proteins on the outer surface of the pathogen. Depending on the type of T-cell, after recognizing the specific pathogen, they are either killing the pathogen (killer T-cells) or signaling to other elements of immune system to attack the pathogen (helper T-cells).

CAR T-cell therapy involves modifying a patient’s own T-cells to express a specific CAR on

their surface. The receptor is designed to recognise antigens commonly found on the surface of cancer cells. To introduce CARs on the outer surface of T-cells, the patient’s T-cells are genetically modified in the lab. A viral vector is often used to knock out the original T-cell receptors and express the CAR construct. The newly created CAR-T-cells are introduced into the patients, where they target and destroy cancer cells expressing the specific antigen for which the CAR is designed.

Incretins

The scientific journal “Science” proclaimed glucagon-like peptide-1 (GLP-1) receptor

agonists The Breakthrough of 2023. These medications, originally approved for type 2

diabetes, demonstrated remarkable weight-loss benefits. GLP-1 is a natural hormone

produced in the intestines that plays a role in regulating blood sugar levels. When we eat a meal, incretins, GLP-1 and Glucose-dependent insulinotropic polypeptide (GIP), are released into the bloodstream. They bind to specific receptors on the beta cells of the pancreas, triggering insulin release. Incretins also suppress the release of glucagon, a hormone that increases blood sugar levels by promoting the breakdown of stored glucose.

GLP-1 receptor agonists are medications that mimic the effects of GLP-1. They bind to the

GLP-1 receptors on pancreatic beta cells, promoting insulin secretion and suppressing

glucagon release. By mimicking the actions of GLP-1, these medications help to lower sugar levels, improve glucose control, and reduce the risk of hypoglycemia. At the same time, they seem to regulate the appetite and delay gastric emptying.

iPSCs

Induced pluripotent stem cells (iPSCs) are becoming a new powerful weapon in lab research. They are a type of stem cell that can be generated from adult cells, such as skin or blood cells, through reprogramming. The process of creating iPSCs involves introducing a set of specific genes into the adult cells. These reprogramming factors reset the adult cells' developmental clock, turning them back into a pluripotent state, similar to embryonic stem cells. Once iPSCs are generated, they can be expanded indefinitely in the laboratory and induced to differentiate into various cell types.

iPSCs are a valuable tool for studying human development and disease, as well as for drug

discovery and regenerative medicine. iPSCs can be derived from patients with genetic

diseases or other conditions, allowing researchers to study disease mechanisms in a dish. By differentiating iPSCs into the relevant cell types affected by the disease, researchers can observe how the disease develops and test potential treatments. Moreover, iPSC-derived cells can screen potential drugs for safety and efficacy. Because iPSCs can differentiate into many different cell types, they provide a more accurate model of human biology than traditional cell culture methods. Finally, because iPSCs can be derived from individual patients, they offer the potential for personalised therapies. iPSCs could be used to generate patient-specific cells for transplantation or to test drugs for individual patients.

Conclusion

These cutting-edge technologies offer unprecedented opportunities for targeted interventions in the treatment of genetic disorders, cancer, diabetes, and a myriad of other diseases. However alongside their immense promise, these biotechnological techniques and therapies also raise important ethical, social and regulatory considerations. The implications of gene editing on human germline cells, the accessibility of advanced therapies, and the long-term safety of these interventions are critical areas that warrant careful attention and thoughtful deliberation. Embracing these innovative techniques with diligence holds the key to unlocking a future where previously incurable conditions become manageable, and where the boundaries of medical possibility are continually expanded.

Written by Matina Laskou

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