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Aspects of Gene Therapy Explained

Gene therapy offers potential treatments for genetic diseases by correcting or replacing mutated genes, but it also poses ethical and safety challenges. There are two main types of gene therapy: somatic, which affects only the patient, and germ-line, which can pass changes to future generations. Stem cells play a crucial role in gene therapy, with embryonic and somatic stem cells having unique properties that can aid in medical treatments.

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32 views2 pages

Aspects of Gene Therapy Explained

Gene therapy offers potential treatments for genetic diseases by correcting or replacing mutated genes, but it also poses ethical and safety challenges. There are two main types of gene therapy: somatic, which affects only the patient, and germ-line, which can pass changes to future generations. Stem cells play a crucial role in gene therapy, with embryonic and somatic stem cells having unique properties that can aid in medical treatments.

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nglmuffiinnss23
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Science, Technology, and Society

LESSON 13: THE ASPECTS OF GENE THERAPY 6) Implications


- Gene therapy holds immense promise for addressing genetic
1) Genes and DNA diseases but presents challenges and potential risks that
- Genes contain DNA, which controls various body functions require further study.
and characteristics, such as hair color, height, and essential
activities like breathing and digesting. The Basic Process

2) Genetic Mutations a) Replacement of mutated gene that causes disease with a


- Malfunctioning genes, often referred to as mutations, can lead healthy copy of the gene
to diseases that traditional treatments cannot cure. b) Inactivation of a mutated gene that is functioning improperly
c) Introducing a new gene into the body to help fight a disease
3) Gene Therapy
- This emerging method has the potential to treat or cure Take note:
genetic-related illnesses by introducing foreign DNA into a • Gene Insertion Process - A gene cannot be directly inserted into
patient’s tissues. a human gene or cell.
- It aims to correct diseases by fixing abnormal genes causing
them. • Carrier or Vector - A carrier or vector is required to insert a gene
into another gene.
4) Historical Context
- Gene therapy was first conceptualized in 1971, inspired by • Common Vectors - Viruses, which are genetically modified to
advancements in recombinant DNA technology. carry normal human DNA, are the most common vectors used.
- It is considered one of the most promising but also • Virus Role - Viruses have naturally evolved mechanisms to deliver
controversial medical fields. their genes to human cells, often in a pathogenic manner (Science
5) Notable Research Daily, 2017).
- In 2015, researchers at Harvard Medical School and Boston Two Types of Gene Therapy
Children's Hospital restored basic hearing in genetically deaf
mice using gene therapy. 1) Somatic gene therapy
- They further improved hearing to a whisper-level (25 decibels) - It involves the manipulation of genes in the patient’s somatic
using an advanced vector, "Anc80," which effectively transfers (body) cells to treat disease but does not pass changes to the
genes to hard-to-reach cells in the cochlea. next generation (Nimsergern, 1988).
2) Germ-line gene therapy The Bioethics of Gene Therapy
- It involves the genetic modification of germ cells (sperm/egg
• How can "good" and "bad" uses of gene therapy be distinguished?
cells) to ensure genetic changes are passed on to future
• Who decides which traits are normal and which constitute a
generations.
disability or disorder?
Stem Cell Gene Therapy • Will the high costs of gene therapy make it available only to
➢ Stem Cells are other cells that have the potential to become any wealthy people?
type of cell in the body. • Could the widespread use of gene therapy make society less
accepting of people who are different?
➢ Stem cells have the ability to self-renew and develop into any type • Should people be allowed to use gene therapy to enhance basic
of cell in the body, such as blood, heart, bone, skin, or brain cells. human traits such as height, intelligence, or athletic ability?

Two Types of Stem Cells Another controversy involves germline therapy. As discussed,
germline therapy is genetic modification of germ cells that will pass the
1. Embryonic stem cells
change on to the next generation.
- It is derived from a four- or five-day old human embryo that is
in the blastocyst phase of development. There are a lot of questions on the effects of gene alteration to the
- The embryos are usually extras that have been created in IVF unborn child and the next generation, since the alteration can be passed
(in vitro fertilization) clinics where several eggs are fertilized in on.
a test tube then implanted into a woman (Crosta, 2013). In the United States, the government does not fund researchers on
human germline gene therapy.
2. Somatic stem cells
- Found in various tissues (e.g., brain, bone marrow, liver, blood,
blood vessels, skeletal muscles, skin) after embryonic
development.
- They remain in a non-dividing state for years until activated by
disease or tissue injury.
- These stem cells can divide or self-renew indefinitely, enabling
them to generate a range of cell types from the originating
organ or even regenerate the entire original organ.

Common questions

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Somatic stem cells are adult stem cells found in various tissues that can self-renew and generate cell types of their originating organ, aiding in regeneration and repair within that specific tissue . In contrast, embryonic stem cells are derived from early-stage embryos, retaining the ability to develop into any cell type in the body, making them potent candidates for a wide range of regenerative therapies and disease treatments . Their roles in regenerative medicine differ in scope, with embryonic stem cells offering broader therapeutic applications due to their pluripotency .

Gene therapy is promising due to its potential to offer cures for genetic diseases that traditional treatments cannot address, but it is controversial due to ethical concerns over genetic modification and its associated risks, including unintended effects and the possibility of gene editing for enhancement rather than treatment . Future advancements, such as improved targeting techniques and comprehensive risk assessments, could mitigate these challenges, enhancing safety and addressing ethical concerns through robust regulatory frameworks .

Balancing the risks and benefits of gene therapy requires comprehensive clinical trials to assess safety and efficacy, alongside stringent regulatory oversight to ensure risks are minimized and benefits maximized . Transparent, informed consent processes are crucial, along with ongoing monitoring for long-term effects and unintended consequences. Ethical guidelines must also be established to prevent misuse, particularly in enhancement cases, ensuring therapy is used responsibly and equitably across diverse populations .

The research at Harvard Medical School and Boston Children's Hospital demonstrated the potential of gene therapy by successfully restoring basic hearing in genetically deaf mice and further improving it to a whisper-level using an advanced vector "Anc80" . This breakthrough highlighted advanced vectors’ effectiveness in targeting hard-to-reach cells and inspired further developments aimed at treating genetic conditions in complex tissues and organ systems .

Viral vectors function by leveraging viruses’ natural mechanisms to deliver genetic material to cells. These vectors are genetically modified to carry normal human DNA instead of their own viral genes, allowing them to insert the desired genetic material into the human genome . Viruses are effective carriers because they have evolved mechanisms to efficiently transfer genetic material into human cells, although typically for pathogenic purposes .

Somatic gene therapy has been explored for treating genetic diseases such as cystic fibrosis, hemophilia, and certain types of blindness . This method is preferred for such conditions because it targets specific body cells without affecting the patient’s germline, ensuring no heritable genetic changes are passed to future generations, thus avoiding the broader ethical issues associated with germline interventions .

The ethical considerations of germline gene therapy include the ability to pass genetic modifications to future generations, raising questions about the long-term effects and potential unintended consequences for the unborn child and subsequent generations . Moreover, the approach is controversial because it involves altering the human germline, with profound implications for individual identity and societal norms, as it could lead to changes in traits considered disabilities or disorders, raising fairness and equity concerns .

Somatic gene therapy involves the manipulation of genes in individual body cells to treat diseases, without passing changes to the next generation . In contrast, germline gene therapy modifies the genetic information in germ cells (sperm or egg cells), ensuring that changes are heritable and affect future generations . These differences influence applications; somatic gene therapy is used for individual treatment, whereas germline therapy has broader ethical and societal implications due to its heritability .

High costs of gene therapy could limit accessibility to only wealthier individuals, exacerbating existing health disparities and prompting ethical concerns about equitable access to life-saving treatments . Such disparities might lead to a societal divide where only affluent individuals benefit from gene therapy advancements, potentially reducing societal acceptance of the technology and raising questions about justice and the fair distribution of healthcare resources .

Gene therapy seeks to correct genetic-related diseases by introducing foreign DNA into a patient’s tissues to fix abnormal genes causing diseases . This approach holds immense promise as it could potentially treat or cure previously untreatable genetic diseases, offering avenues to address a wide range of conditions. However, it also presents challenges and potential risks, requiring further research to understand unintended consequences and long-term effects .

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