From: mk_thisisit
Recent developments indicate a significant shift in medical treatment, with countries like Great Britain and the United States giving “the green light” to therapeutic methods based on genome editing [00:00:09] [03:50:00]. This marks a major breakthrough in the field of medicine [04:11:00].
What is DNA and Genome Editing?
DNA is described as the “essence of life” [00:46:00] [05:05:00]. It defines each individual, their appearance, certain behaviors, and predispositions [05:07:00].
At present, scientists can significantly interfere with the human DNA strand [01:11:00]. This includes:
- Modifying DNA sequences by introducing fragments that were not originally there [01:21:00].
- Deactivating certain genes to make them inactive [01:30:00].
- Amplifying or duplicating fragments of sequences [01:39:00].
- Intentionally damaging DNA to alter its function [01:46:00].
Genome editing, specifically, is based on the intentional induction of specific types of damage in very precise places within the DNA sequence [08:51:00]. Understanding how the DNA repair machinery works is crucial for properly designing these editing “machines” to ensure effectiveness [09:03:00].
Current Therapeutic Applications
Therapeutic methods based on genome editing are now being implemented for diseases like sickle cell anemia, marking a significant breakthrough [04:07:07] [04:11:00] [04:13:00]. This is the first time in history that such therapies have been accepted and will be implemented in patients on a large scale in Great Britain and the United States [01:09:00] [01:11:00] [01:15:00] [01:18:00].
Regarding oncological diseases, certain drugs from the DDR (DNA Damage Response) group, which inhibit the repair of DNA damage, have been on the market for many years [01:15:00] [01:16:00] [01:17:00] [01:19:00]. Examples include PARP inhibitors (e.g., Olaparib, Niraparib), used for ovarian, breast, and prostate cancer [01:17:00]. These drugs aim to damage DNA precisely in cancer cells without harming normal cells, thereby minimizing side effects compared to traditional chemotherapy [01:13:00]. Another type of drug being tested is radiopharmaceuticals, which emit radiation to cause DNA breaks specifically in tumor cells [01:42:00].
NOTE
Dr. Magdalena Kordon shares a personal testament to the efficacy of these treatments, stating her mother is alive and disease-free after three years thanks to such therapies [01:48:00].
Challenges and Ethical Considerations
Despite the technological advancements, significant challenges remain, particularly concerning the ethical and evolutionary implications of human genome modification [00:18:00] [02:22:00]:
- Evolutionary Impact: It is difficult to assess the long-term evolutionary consequences of large-scale interference with the human genome [02:27:00]. There are concerns about how such modifications will affect future generations and the further development of the human species [02:37:00].
- Defining Humanity: A Polish writer, Jacek Dukaj, predicts that future generations might be so genetically modified that they could no longer be called Homo sapiens in the traditional sense [03:00:00].
- Embryo Modification: The ability to modify human embryo DNA in the mother’s womb to cure diseases like Down Syndrome is a complex issue, deemed “not that simple” due to ethical and safety concerns [02:04:00] [02:14:00] [02:55:00].
The Role of Dr. Magdalena Kordon and IntoDNA
Dr. Magdalena Kordon, a biophysicist from Jagiellonian University and co-founder of the startup “intoDNA,” is a key figure in this field [01:01:00] [01:03:00]. Her doctorate focused on understanding how human cells protect DNA by maintaining its stability and security, specifically investigating single-strand DNA breaks and the protein XRCC1 involved in their repair [05:54:00] [07:21:00].
Cells have developed a set of tools to prevent DNA damage and quickly repair it when it occurs [07:08:00]. DNA is constantly exposed to external and internal factors that can cause it to break or become damaged [06:28:00]. Damage can include single- and double-stranded breaks, errors in the genetic code, or attached structures that impair DNA function [06:37:00].
Dr. Kordon's doctoral research revealed that the XRCC1 protein is delivered to the site of a single-strand break like a "tanker," parking a "set of tools" at full readiness to deliver molecules needed for repair [07:54:00].
The startup “intoDNA” develops and applies the “stright technological platform” [01:26:00]. Born out of research at Jagiellonian University, this platform addresses the previous lack of technology for detecting DNA damage directly at a low level [01:29:00]. Their technology allows for:
- Precise definition and counting of mechanical DNA damage [01:35:00].
- Localization of damages and determining their quantity [01:15:00].
- Assessing if a patient will respond to a specific type of targeted therapy or if treatment needs to be adjusted [01:42:00].
IntoDNA’s primary goal is not to develop therapies directly but to assist companies and laboratories worldwide in developing new generation targeted therapies [01:11:00]. Their focus is mainly on oncology, but also extends to neurodegenerative diseases (e.g., Huntington’s disease) and other areas where DNA damage or stability is crucial [01:31:00].
The Future of Medicine and Humanity
The ability to modify human DNA opens up possibilities for the future of medicine and humanity:
Personalized Medicine
The speaker believes the “only right direction” for oncological therapies is personalized medicine [02:06:00]. This involves:
- Very accurate diagnostics of the patient [02:11:00].
- Characterization of a specific tumor, including genetic and functional testing [02:16:00].
- Tailoring therapy to the needs of a specific individual patient [02:41:00].
- Genome editing therapies could be one solution within this personalized approach [02:51:00].
Curing Cancer
While a single “cure for cancer” does not exist due to the unique nature of each tumor [01:56:00], there is strong belief that cancer will cease to be a fatal disease in many cases through advanced personalized therapies [01:23:00].
Extending Lifespan
There is a strong possibility that through modifications of DNA, humans may eventually exceed the age of 100 [02:04:00] [02:06:00]. Historically, continuous improvements in quality of life and treatment effectiveness have led to a steady increase in average human survival rates [02:14:00]. While DNA largely determines species lifespan, individual predisposition to diseases is also written in the genome [09:14:00] [09:27:00]. The stability of DNA strands and the effectiveness of repair mechanisms decrease with age, but lifestyle choices can mitigate this [09:56:00].