Prepared to become the foundation of the health care system of the future (1-3)

Gene therapy. A new era of hopes, dreams and fears

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Genomics has put humanity on the cusp of a new era of hopes and dreams to eradicate devastating diseases and genetic disorders such as cancer, thalassemia, muscular dystrophy, cystic fibrosis and sickle cell anemia.

Genomics emerged as early as the sixties of the last century, as scientists speculated that DNA sequences could be inserted into patients' cells to treat genetic disorders.

In the early eighties, it was confirmed that one could use a virus to insert genes into hematopoietic stem cells.

In 2003, the Human Genome Project was completed, giving us a complete blueprint of our DNA.

In the past decade, gene therapy has become a reality for many diseases, especially those caused by mutations in a single gene.

After a difficult start, the new treatment has attracted widespread attention and attracted biopharmaceutical industry in many countries of the world.

As the UAE enters the global race to monitor the prospects of this science, specialists emphasize the need to find controls and legislation that keep pace with the great development in the uses of genomics, to protect "genetic privacy".

The positive attitude of religious and biomedical scientists towards genetic research and therapeutics paved the way for large-scale genomics projects in Qatar and Saudi Arabia in 2013.

The UAE, Bahrain, Oman, Kuwait and Egypt have also begun announcing their genomics initiatives.

The process of developing genomics policies and guidelines is still in its infancy in these countries with no concrete positions on how to regulate the editing of the human genome as existing and already planned genomic projects in the Muslim world progress.

Religious scholars and specialists disagree not only on the ethics and governance of human germ lineage, but also on techniques aimed at altering the composition of DNA.

Some generally believe that the human genome should not be attempted to be edited for reproductive purposes at this time, but studies should continue that would make gene therapy safe and effective, while others want to move forward with this, and most stakeholders agree that public discussions should be held and allow the public to decide whether germline modification is allowed or not.

Dr. Mohammed bin Ahmed bin Marwan Al-Zahrani, Senior Specialist in Genetic Medicine and Pharmacogenomics from Saudi Arabia, said: "Gene editing technology is one of the world's most popular applications. It is a technique that allows gene editing in organisms in general, including plants, animals and humans. However, most of its applications are currently being implemented in the agricultural field, to improve selected plant strains, develop more productive agricultural crops, adapt to climatic conditions, the nature of the land and its suitability for agriculture, in addition to developing mechanisms to resist diseases and pests.

As for interfering in modifying or changing the human or animal genomic content, there are major legal and ethical concerns that necessitated the establishment of strict controls and laws for it."

He stressed that there is strict control over research, studies and experiments conducted on animals, due to the sensitivity of genetic modification and its unexpected effects, which may appear on later generations.

Al-Zahrani also stressed that areas of intervention for genetic editing must be accurately identified, while continuing to work on experiments and monitoring impacts across generations to avoid any tampering with biodiversity and ecological balance, "especially as we put improving the quality of life first."

Dr. Ahmed Abutyoun, Director of the Genomics Centre at Al Jalila Hospital in Dubai, Assistant Professor of Genetics Studies at Mohammed Bin Rashid University of Medicine and Health Sciences, said that the use of genomics in treatment in general is divided into several categories, including "genetic scanning", and is used to study and analyze the genes of a group of individuals in order to determine the likelihood of future diseases, and then work to prevent them. "Gene therapy," in which gene therapy or drug is used to treat and eliminate a particular disease. "Genetic editing", through which specific genetic modifications are made to people or embryos in order to give them specific genetic characteristics.

Abu Tayoun stressed the need for legislation and laws regulating and controlling the uses of genomics in general, to ensure that the therapeutic purposes targeted by this science do not deviate to other purposes that violate legal and ethical controls in society, such as using it to have births with special specifications, or for recreational purposes far from therapeutic necessities. He continued: «The most important thing related to gene therapy is the privacy of patient information», stressing the need for laws and legislation to include this aspect to ensure the preservation of this data, especially since many pharmaceutical companies may seek to obtain them for the purpose of producing special drugs based on obtaining detailed genetic information about diseases. He stressed the need to apply a set of ethical controls, as this science allows the possibility of modifying the genes of embryos, before fertilization of the egg, to acquire specific formal and mental specifications, which must be controlled by laws and legislation so as not to misuse them later and leave them to the whims of people without regulation or control.

He pointed out that «gene therapy heralds a new era in the history of medicine, where we pilgrimage in the treatment of dangerous incurable diseases, most notably spinal muscular atrophy through an injection containing gene therapy, and also allowed to predict the possibility of developing specific diseases, and then avoid them through periodic examinations and various methods of prevention».

Dr Mark Haber, associate professor at the Institute of Cancer and Genetic Sciences at the University of Birmingham Dubai, said: "Gene therapy offers high hopes for many patients, but requires extreme caution in harnessing its transformative potential, as it involves two key aspects: the technical dimension, where the current gene therapy quality generates concerns about unintended modifications in our DNA, and the ethical dimension, which relates to drawing limits to permissible genetic modifications."

"Gene therapy is poised to become the foundation of the healthcare system of the future," he said, adding that "gene therapy is the pinnacle of medical achievement after we sequenced human genes and then found ways to modify them."

He pointed out that gene therapy has contributed to the eradication of diseases such as cystic fibrosis and hemophilia and the treatment of diseases common in the Middle East such as sickle cell anemia and thalassemia, in addition to the hope of treating some types of cancer and diseases such as diabetes and heart disease. "With that great potential in place, it's time to think about our definition of what counts as a disease, assess our perspective on unwanted human traits, and consider the complex task of prioritizing and selecting traits that we want to eliminate from the human genetic diversity group."

A doctor specializing in genomics and genetics research from the UAE, who preferred not to be named, said that genetic editing today announces a new race, as many countries try to be leaders in new technology.

He added that gene therapy could be a turning point in the history of medicine, but genome editing is one of the most promising works of science for humanity: "Gene therapy alone cannot provide a quick solution to diseases that depend on many genes, as well as environmental factors and lifestyles, but it can be used to treat diseases caused by a defect in a single gene."

Aisha Mohammed al-Mulla, a member of the Federal National Council, asks how the regulatory landscape of genomics and its new applications and tools evolve in response to technological development.

Emphasizing the rapid development in understanding of the human genome in recent decades, she said, "However, there is still a lot to learn about associated processes, such as gene regulation and how gene editing can affect them. However, this concern is not limited to genome and its tools as a science, because there are other considerations, such as: uncertainty about non-targeted impacts, governance and regulation of safety-related issues, and misrepresentation of expectations and speculation."

Al Mulla stressed the potential and significant benefits of gene therapies, which justify innovation and research, although the regulatory framework is still under public debate.

"Rapid advances in innovative therapies that are highly personalized, such as gene editing, defy the ongoing framework and guidelines. "As new genomics technologies evolve, it will be desirable to demonstrate a governance framework in the future, requiring regulation to be directed to an active field rather than a specific technology, where new tools will inevitably continue to be developed for the same intended purpose and potential applications."

"Focusing on current genomics technologies risks exclusively developing frameworks that will become obsolete in a very short time. For example, the UK regulation covering gene therapies has been drafted extensively, focusing on the intended purpose rather than the specific technology used. Therefore, the genome editing of somatic cells is currently being regulated within the framework of current gene therapies, as an advanced therapeutic medical product."

Dr. Maryam Mattar, President of the UAE Genetic Diseases Society, stressed the importance of the need for laws protecting genetic testing data, as tests reveal sensitive personal information related to genetics and individual health, and can have significant impacts on the people concerned and their lives. She said genetic testing data is highly protected because of its sensitive and personal nature, and if such data is disclosed or used illegally or undesirably, individuals may be exposed to discrimination, exploitation, psychological harm or invasion of their privacy. For example, genetic data can be used to determine people's health orientations and concerns or to determine their genetic desires, which can negatively affect their career, insurance or even personal relationships. "Genetic testing data protection laws are essential to ensure that individuals' privacy is preserved and protected from exploitation or abuse. These laws typically include requirements to obtain the explicit consent of individuals before collecting their genetic data, ensuring the confidentiality of this data and not sharing it with unauthorized parties, setting time periods for data retention and destruction after the specified period expires, and implementing strong security measures to protect this data from unauthorized penetration or access.

The World Health Organization (WHO) released two UN reports in 2021, which point to a "leap forward" in the regulation of DNA technology after extensive global consultations on various types of gene therapies.

• Gene therapy has contributed to the eradication of cystic fibrosis and hemophilia and the treatment of common diseases such as sickle cell anemia and thalassemia.

Gene therapy could be a turning point in the history of medicine, but genome editing is one of the most promising works of science for humanity.

Gene therapy alone cannot provide a quick solution to diseases that depend on many genes, environmental factors and lifestyles.

There is still a lot to learn about the processes involved, such as gene regulation and how gene editing can affect them.

• Focusing on current genomics technologies risks developing frameworks that will become obsolete in a very short time.

With advances in genomics. We got a different healthcare landscape, where we can practically participate in understanding and shaping our personal health profiles.

However, we need to balance the prospects of this science with the challenges posed by the technological, financial, ethical, and cultural constraints to which it is subject.

"Emirates Today" opens the file of development in understanding this event, and understanding public views on the editing of the human genome and foreseeing the future in it by surveying the opinions of experts and specialists from the UAE and Arab countries, in addition to polling parents with children of determination, by relying on an electronic survey to explore people's attitudes towards the impact of gene technology on them and their communities.

The use of science in what works

Dr. Ahmed bin Abdulaziz Al-Haddad. From the source

The Chief Mufti, Director of the Iftaa Department at the Islamic Affairs and Charitable Activities Department in Dubai, His Eminence Dr. Ahmed bin Abdulaziz Al-Haddad, said that «the use of the human genome in treatment and improvement, is taking rapid steps, reaching the extent of what is called genetic engineering or genetic modification, which means: direct impact on the genetic material of man or animal, and this may be with the intention of tampering with man according to whims, if this engineering is used to tamper with the human being created by God Almighty in the best calendar, then he will be just, and honor him by Distinguish it from the rest of creation, which enables him to succeed on earth to know God Almighty by faith and worship him with what he legislated on the tongues of his messengers, peace and blessings be upon them, and the architecture of the earth that he harnessed to man to benefit from its bounties, and Amr the truth that this is the one that Satan pledged to seduce the children of Adam, and God Almighty warned his servants of it, and he said: {And to their princes, let them change God's creation}, this is what he pledged and was able to do in those who liked him and followed him. Since it is a matter in man to distinguish good from evil and harmful from beneficial, God Almighty has warned him of this temptation by saying: {And whoever takes Satan as a guardian without God has lost a clear loss}, and here is the pledge of Satan is realized today because of this technological revolution, which was originally used to benefit humanity not to harm it.

He added that «cohabitation Muslims seek to develop science and use it in what benefits not in what harms, so the use of the human genome must be a useful use is harmless, in order to know genetic diseases and what can be treated, not in the so-called hybridization or genetic improvement, or otherwise, this is not permissible Sharia or reason, and while science authorizes that, religion and ethics must control science to be useful and harmless», pointing out that «this topic was Present in jurisprudence academies from a long time, the International Islamic Fiqh Academy thoroughly researched it in courses

Several decisions were adopted at the 21st session in Riyadh and the 24th session in Dubai, stipulating in the first decision No. 203 that it is permissible to use the human genome or part of it in beneficial fields, because of the interests that Sharia has urged to obtain, such as the prevention and treatment of diseases.

He added that «in this case the award stipulates that all genetic diagnoses preserved or prepared for research purposes or for any other purpose must be completely confidential, and they are not disclosed except in the cases specified in the Academy's decision No. 79 (10/8) on the confidentiality of the medical professions, and the decision No. 142 (8/15) on the doctor's guarantee. It stipulates that the doctor must obtain the patient's consent to disclose his secret to his family if he is seriously ill, and if the patient does not agree to this, the doctor must try to convince him to agree in order to ensure the lives of others in his family. The decision stipulates the need to obtain valid and legitimate permission from the person himself or his legal guardian to analyze his genetic map, while taking care of the interest of the person concerned. As for anything that is contrary to morality or in which it is tampering with human dignity, it has been decided that it is not permissible to use the genome in a harmful way or in any way that violates Islamic law."

"No research, treatment or diagnosis related to a person's genome may be carried out except after a prior and thorough assessment of the potential risks and benefits associated with these activities, while adhering to the provisions of Islamic law in this regard," he said.

5 Challenges

Specialists have identified five main challenges facing gene therapy globally:

1 "Delivery technology": Gene therapy requires the delivery of modified genes to target cells in the body, and this technology faces challenges related to the effectiveness and safety of delivering genes to the appropriate places within the body, and ensuring that the modified genes reach adequately and effectively to the target cells.

2 "Safety challenges": Gene therapy techniques must be developed with caution to ensure patient safety. Unwanted activation of genes other than target may occur, leading to dangerous side effects. Extensive clinical studies should be conducted to evaluate the safety and efficacy of gene therapy before it is used in patients.

3 "Regulation and law": There may be challenges in putting in place laws and regulations to control the use of gene therapy. A legal and ethical framework must be developed to deal with issues such as genetic privacy, genetic modification in embryos and the public presentation of the technology.

4 "Ethics": Many controversial ethical issues are raised around gene therapy, including genetic specificity, gene editing in embryos and its impact on future generations, and the distribution of gene therapy between individuals and communities.

5 "Financial Cost": The high cost of gene therapy is one of the biggest challenges it faces.