From: mk_thisisit
The field of medicine is rapidly advancing, with significant breakthroughs in the modification of DNA for therapeutic purposes. Recent developments, particularly the regulatory approval of genome editing methods in countries like Great Britain and the United States, mark a pivotal moment in treating various diseases [00:00:09] [03:50:00].
The Essence of DNA and its Significance
DNA is considered the essence of life, defining each individual, including appearance, certain behaviors, and predispositions [05:07:07] [05:19:19] [05:39:00]. While human intelligence doesn’t have a direct DNA translation, certain genes encode proteins that influence brain and nervous system efficiency, indicating an indirect link [04:43:00] [04:57:00]. The stability of DNA and the effectiveness of its repair mechanisms also influence how long an individual lives and their susceptibility to diseases [09:14:00] [09:56:00].
Understanding DNA Damage and Repair
DNA is a molecule present in almost every cell, constantly exposed to external and internal factors that can cause damage, including single- and double-strand breaks, errors in the genetic code, or attached structures that impair function [06:16:00] [06:33:00]. Human cells have evolved a comprehensive set of tools to prevent and rapidly repair such damage [07:08:00].
Dr. Magdalena Kordon’s doctoral research, conducted at the Jagiellonian University, focused on understanding how cells protect DNA by maintaining its stability and security, specifically investigating single-strand DNA breaks [05:54:00] [07:21:00]. Her work shed light on how the XRCC1 protein, a key factor in repairing these breaks, is delivered to the damage site [07:30:00] [07:54:00]. Understanding these natural repair mechanisms is critical for designing effective genome editing tools, which intentionally induce specific types of damage in precise locations to facilitate therapeutic interventions [08:51:00].
Therapeutic Applications of DNA Modification
Cancer Treatment
DNA modification is already implemented in treating oncological diseases [00:16:00]. The goal is to achieve a targeted approach that minimizes side effects associated with traditional chemotherapy [16:15:00].
Current and developing methods include:
- Genome Editing: While not yet widely commercialized for cancer, techniques like Crispr are groundbreaking [15:27:00]. They allow for the intentional induction of DNA damage in specific cancerous cells or the modification of their DNA sequence, with tools like intoDNA’s STRIGHT platform able to verify if these edits are occurring correctly [08:51:00] [15:44:00].
- DDR (DNA Damage Response) Therapeutics: These drugs, also known as DD therapy or Damage Response therapeutics, inhibit the repair of DNA damage specifically in cancer cells, leading to their destruction [16:06:06] [16:31:00]. PARP inhibitors (e.g., olaparib, niraparib) are examples that have been on the market for many years, showing very good effects in treating ovarian, breast, and prostate cancers [18:14:00].
- Radiopharmaceuticals: These molecules are precisely introduced into the patient’s body, attaching to tumor cells (but not normal cells) and emitting radiation (e.g., Alpha or Beta therapy). This radiation causes DNA breaks specifically in cancer cells, leading to their demise [16:42:00] [17:12:00].
The vision for treating cancer is increasingly shifting towards personalized medicine, involving accurate diagnostics, genetic testing, and functional characterization of individual tumors to tailor therapies to each patient’s specific needs [20:06:00] [20:41:00].
Other Diseases
Beyond cancer, therapeutic genome editing has seen its first major breakthrough with the approval for treating sickle cell anemia [17:40:00]. DNA stability and damage are also crucial in neurodegenerative diseases, including Huntington’s disease, making them another area of focus for DNA-based therapies [11:39:00] [11:46:00].
Ethical and Evolutionary Considerations
Interfering with the human genome on a large scale presents significant challenges, particularly in assessing its long-term evolutionary consequences on future generations and the development of the species [00:18:00] [02:22:00]. Beyond technical capabilities, ethical and safety issues are paramount [02:48:00]. Some futuristic visions, such as those proposed by writer Jacek Dukaj, suggest that future generations might be so extensively genetically modified that they may no longer be considered Homo sapiens in the traditional sense [03:10:00].
Despite these concerns, the continuous improvement in quality of life and treatment effectiveness suggests that humans may eventually be able to exceed the age of 100 through such modifications [24:11:00].
intoDNA: Accelerating New Generation Therapies
Dr. Magdalena Kordon is a co-founder of the startup intoDNA, which originated from research at the Jagiellonian University [01:03:00] [11:51:00]. The startup focuses on helping companies and laboratories worldwide develop new generation targeted therapies, primarily in oncology and neurodegenerative diseases [11:12:00] [11:31:00].
intoDNA’s core technology is the STRIGHT technological platform, developed to precisely detect, count, and localize mechanical DNA damage, even at very low levels (e.g., a few cracks in a specific place) [12:29:00] [13:04:00] [13:17:00]. This high sensitivity and specificity were lacking in existing technologies [13:04:04] [13:19:00]. By understanding the type and extent of DNA damage, intoDNA can help predict patient response to specific drugs and optimize treatment strategies [14:35:00]. Their work aims to accelerate the development process of new therapeutics, which can otherwise take over a decade, by providing crucial precision and effective decision-making support [22:01:00].