The Difference Between Somatic and Germ Line Gene Therapy

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The Difference Between Somatic and Germ Line Gene Therapy

Somatic gene therapy is a treatment for genetic defects that involves inserting new DNA or RNA into the body of someone suffering from disease. This corrects the mutated gene, thus eliminating its cause for the condition. From an ethical perspective, this type of treatment seems relatively uncontroversial.

Germ line gene therapy, on the other hand, is a much more controversial method for treating genetic diseases. This procedure involves inserting desired genes into germ cells – reproductive cells in humans such as egg and sperm cells that create children – during early stages of embryonic development (e.g. during in vitro fertilisation) or after birth when the child is born.

This type of therapy has the potential to be highly effective in curing genetic diseases that are inherited or acquired. Unfortunately, it is currently illegal in many countries due to ethical concerns and it remains uncertain whether future generations will be protected from any long-term repercussions from this type of treatment.

The ethics of germline editing are once again being debated with the advent of Clustered Regularly Interspaced Short Palindromic Repeats-associated (CRISPR) technology, which may enable efficient and highly targeted genome editing in single cell embryos. This potential breakthrough brings human germline editing back into legal and ethical discussions once again.

Scenarios 1 and 2 involve editing a specific mutation in the CFTR gene locus within single-cell embryos with the goal of curing cystic fibrosis (CF). In scenario 1, no genetic modification would affect the embryo phenotypically, while scenario 2 eliminates HIV risk for both the developing embryo and its offspring.

In scenario 2, genetic modification is achieved through DNA-recombination, a form of gene editing known as DNA-recombination. Here, an inactive transgene cassette carrying the wildtype CFTR gene is flanked by DNA recognition sites and removed upon activation by CRISPR-Cas in all germ cells. This ensures that modified embryos would phenotypically cure themselves of CF while their offspring will not carry this recombinant transgene cassette since it has been physically removed from the nucleus after recombination.

Genome editing is an incredibly powerful and versatile tool, offering new opportunities to cure genetic diseases. Additionally, it could potentially be employed for crop improvement, age-related disorders and even creating entirely new organisms.

The legitimacy of germline editing in single-cell embryos, as proposed here, is particularly pertinent since it’s usually assumed that such interventions are justified based on a secured genetic diagnosis indicating medical need and established gene therapy. If these assumptions aren’t fulfilled – for instance by implanting an edited embryo into an unwell pregnant woman – then such germline intervention might not qualify as therapeutic and could pose major challenges to existing regulations regarding acceptable risks associated with germline interventions like assisted reproduction procedures (Coutts 1998).

In scenario 2, gene editing is done through CRISPR-Cas, an efficient and highly targeted technique for single cell embryo genome editing. CRISPR-Cas has several advantages over other genome editing methods since it requires only minimal modifications to the cell nucleus with few side effects. As such, this procedure may be better suited for genetically important genes than other methods such as somatic gene editing or recombination.

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