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Is Gene Editing Allowed in the US?

Published in Gene Editing Regulation 3 mins read

In the US, the allowance of gene editing is nuanced, varying significantly based on the type of cells being modified and the intended purpose. While certain applications, particularly those involving heritable changes in human embryos, face strict prohibitions, other forms of gene editing, such as those targeting somatic (non-reproductive) cells, are permitted under rigorous regulatory oversight.

Understanding Gene Editing in the US

Gene editing broadly refers to a set of technologies that can cut DNA at a specific spot. This allows for precise changes to the DNA sequence, such as adding, removing, or altering genetic material. The key distinction in the US regulatory landscape lies between somatic cell gene editing and germline (heritable) gene editing.

Somatic Gene Editing: Generally Permitted and Regulated

Somatic gene editing involves making changes to the DNA of non-reproductive cells (e.g., blood cells, muscle cells, brain cells). These changes are not passed down to future generations. This form of gene editing is primarily pursued for therapeutic purposes, such as treating genetic diseases like sickle cell anemia, cystic fibrosis, or certain cancers.

  • Therapeutic Focus: Most approved or clinical-stage gene therapies fall into this category, aiming to correct genetic defects in affected individuals.
  • Regulatory Pathway: These therapies are subject to stringent review and approval processes by the Food and Drug Administration (FDA), which assesses their safety and efficacy through multiple phases of clinical trials.
  • Examples: Treatments like Zynteglo for beta-thalassemia and Casgevy for sickle cell disease are examples of FDA-approved somatic gene therapies.

Prohibitions on Heritable (Germline) Gene Editing

Conversely, germline gene editing involves making changes to the DNA of reproductive cells (sperm or egg) or early embryos. Crucially, any genetic modifications made at this stage would be heritable, meaning they would be passed down to all future generations of an individual's offspring.

The US has significant restrictions concerning heritable human gene editing:

  • FDA Clinical Trial Ban: The Food and Drug Administration (FDA) is explicitly barred from considering any clinical trial application "in which a human embryo is intentionally created or modified to include a heritable genetic modification." This effectively prevents any clinical use of gene-edited human embryos with changes that could be inherited.
  • NIH Funding Ban: The National Institutes of Health (NIH) is prohibited from using federal funds to support research that involves "human germline editing." This means federally funded institutions cannot conduct research that aims to alter genes in sperm, eggs, or embryos in a way that would be passed on through inheritance.

These prohibitions reflect ethical concerns about altering the human germline, including potential unintended consequences for future generations, the concept of "designer babies," and issues of consent for individuals not yet born.

Summary of US Gene Editing Landscape

The table below summarizes the current landscape of gene editing allowance in the United States for human applications:

Type of Gene Editing Affected Cells Inheritable? Status in the US Key Regulatory/Funding Landscape
Somatic Gene Editing Non-reproductive (e.g., blood, muscle, brain) No Generally Permitted for therapeutic use Regulated by FDA; requires extensive clinical trials for safety and efficacy.
Germline Gene Editing Reproductive (sperm, egg) or Embryo Yes Strictly Prohibited for clinical use and federal funding FDA barred from reviewing clinical trials involving heritable modifications in human embryos. NIH barred from funding human germline editing research.

In conclusion, while somatic gene editing for therapeutic purposes is a growing field under strict FDA regulation in the US, heritable (germline) gene editing in humans is not allowed for clinical applications and is barred from federal funding due to profound ethical and societal considerations.