Understanding Gene Therapy

Gene Therapy Definition

• According to the Human Genome Organization (HUGO) Ethics Committee (2001), gene therapy is defined as the correction or prevention of disease through adding and expressing genetic material.
• This process aims to reconstitute missing genetic functions, correct aberrant ones, or interfere with disease-causing processes.

Challenges of Gene Transfer

• Introducing new genetic material using a vector like a virus.
• Targeting the material to the correct part of the body.
• Ensuring the new material functions correctly.

Evolution of Gene Therapy Focus

• Initial focus was on single-gene disorders like Huntington’s disease.
• Current interest includes multifactorial conditions like breast cancer, which involve multiple genes and environmental triggers.

Genes and Multifactorial Conditions

• Genes might increase susceptibility, but not guarantee the development of multifactorial conditions.
• Lifestyle changes may be more effective than gene therapy in certain cases.
• Gene therapy can potentially cure genetic disorders, while other treatments only alleviate symptoms.

Key Distinctions in Gene Therapy Debates

• Somatic vs. Germline gene therapy is a key distinction.
• Therapy vs. Enhancement is another key distinction in the issue

Somatic vs. Germline Gene Therapy

• Somatic gene therapy targets somatic cells (body cells) of an individual.
• Germline gene therapy targets reproductive cells, affecting future generations and the gene pool.
• Somatic therapy, like other treatments, may increase the concentration of mutant genes by enabling affected individuals to reproduce.

Germline Therapy Potential

• Germline therapy has the potential for greater control over and impact on the gene pool than somatic therapy.

Therapy vs. Enhancement

• "Therapy" is the treatment of disease.
• "Enhancement" employs therapeutic techniques to go beyond disease treatment and introduce improvements.
• Interventions can be therapeutic and have enhancement effects.

Challenges and Setbacks

• Early gene therapy faced disappointments due to low success rates.
• The death of Jesse Gelsinger in 1998 raised concerns, leading to renewed debates about ethics, regulations, and conflicts of interest.

Alternative Biomedical Research

• RNA interference (stopping gene expression), stem cell research, and nanomedicine are also attracting attention as alternatives.
• These new technologies can offer more effective delivery systems for gene therapy.

Continued Research

• Despite setbacks, gene therapy is still being discussed and researched.
• In April 2008, there were reports of successful gene therapy trials for a rare form of blindness.

Ethical Considerations

• Relationship between gene therapy and personal identity.
• Individual choice vs. public health.

Somatic Gene Therapy Argument

• Genetic disorders cause suffering, and somatic gene therapy offers a potential cure.
• It is considered similar to other medical interventions like organ transplants.

Ethical Constraints on Somatic Therapy

• Somatic therapy should be subject to ethical constraints like informed consent and confidentiality.
• The Clothier Committee considered somatic therapy as research involving human subjects, subject to regulations.
• The Committee emphasized the obligation to inquire and study ethically, ensuring patient interests prevail over the pursuit of knowledge.

Potential Benefits of Germline Therapy

• Extends medical benefits not only to the existing patient but also to their descendants.
• Aligns with considerations of reproductive autonomy and welfare of future people.

Concerns with Germline Therapy

• Germline therapy raises new issues of principle because it affects future generations without their consent.
• Reproductive autonomy includes freedom to make decisions about whether to reproduce and the qualities of children.
• Disagreements persist on whether reproductive autonomy should include the freedom to decide on the qualities of the children.

Reproductive Choice

• Should people be allowed to choose to have children with genetic disorders, such as deaf parents wanting a deaf child?
• Reproductive autonomy may have limits based on the interests of future children.
• Some argue that being deaf can be in someone's interest, such as being part of a deaf community.

Preimplantation Diagnosis and Embryo Selection

• Techniques allow couples to produce a "winning combination" of genes without germline therapy.
• Cell nuclear replacement can be used to avoid mitochondrial diseases by transferring the nucleus from the woman's egg to a donor egg.
• This results in a genetic change that is passed on to future generations, which is a form of germline therapy.

Opposition to Three-Parent Children

• Some are concerned that cell nuclear replacement results in children with one father and two mothers, and one of the mothers contributes the egg with mitochondrial DNA.
• Others downplay the role of mitochondrial DNA, comparing it to a battery in a radio.

Concerns of Mutant Mitochondria Transfer

• There are safety concerns about the possibility that some mutant mitochondria might be transferred with the nucleus and cause disease in future generations.

Options for People with Mitochondrial Disease Concerns

• Avoiding reproduction entirely.
• Accepting the risk of serious disorders in their offspring.

Moral Boundary

• Therapy and enhancement is used in somatic and germline therapy to establish a moral boundary.

Concerns about Genetic Interference and Control

• Boundaries provide reassurance about a slippery slope toward unacceptable genetic interference and control.

Media Coverage of Designer Babies

• Couples enhance their children to suit their whims and high expectations
• This is undesirable.

Altered Status of Resulting Person

• Being the result of someone else's design, an artifact, fundamentally alters one's status and self-understanding as a human being.
• Critics suggest that enhancement leads to losing something essentially human.

Ronald Dworkin's Examination

• Examines the meaning of "sacred," noting widespread belief in the intrinsic value of human life.
• Different interpretations of this value exist in gene therapy.
• Dworkin argues that the value of evolved plant species differs from genetically engineered ones.

Societal Perception

• Engineered species are valued differently from unengineered ones due to germline therapy concerns.
• Gene therapy aims to extend life or enhance sports capabilities and is gaining support.

Criticisms on the control of abuse

• The genetic basis of characteristics like intelligence should not be researched.
• Individual choices can be influenced.
• Cumulative effects are significant.

Resource allocation concern

• Related to the candidate selection of treatment in the controversy use of gene therapy and curing disease.

Feared Reinforcement of Unequal Differences

• Enabling the wealthy to acquire favorable genes creates a gap.
• Differences are based on developed societies verses developing countries.
• Determined by how genes against the environment influence in determining an individuals' life chances.

Conclusion

• Research on the extent of human variation, the complicated nature of genetic and environmental factors undermines the symbolic importance of "gene" and the search for gene-based therapies continue.