House Medicaid and Behavioral Health Appropriations Subcommittee May-June 2025: spending the Opioid settlement, MI Primary Care, Mosaic counseling

Answer: b, the presence of different genetic cells. The implications for life are huge.

This backgrounder from the Witherspoon Institute's journal, Public Discourse, spans the gamut of MHF Forum topics. Federal NIH research, Michigan Rare Disease Council and this Mosaic bill, industry research and prenatal diagnostics, and of course the foundation purpose of healthcare to support life.

https://www.thepublicdiscourse.com/2026/01/99931/

The MAHA Case for Advanced Genetic Editing

Emma Waters    |    January 13, 2026

If the goal of medicine is to protect and restore life, then our efforts and investments should flow to therapies that treat the sick, not to technologies that eliminate them before they are born.

When you think of the “Make America Healthy Again” (MAHA) movement, you might picture fertile soil, organic farms, and homeopathic remedies. For some, that vision is a welcome change; for others, it may seem like a rejection of modern science. But the next frontier in root cause health care is not on farms at all. Indeed, it is taking shape in cutting-edge gene and cell therapy labs across the country, where researchers are tackling diseases at the cellular level with personalized treatments aimed at curing life-threatening conditions, like sickle-cell anemia and Huntington’s disease, and CAR-T therapy, a personalized immunotherapy that uses a patient’s own immune cells to treat certain cancers.

Under Robert F. Kennedy, Jr., the Department of Health and Human Services has advanced innovations in gene and cell therapies. In March of last year, the administration hosted a roundtable discussion with leading biotechnology groups. By June, Secretary Kennedy, surgeon Marty Makary, economist Jay Bhattacharya, and Dr. Mehmet Oz had convened an FDA roundtable to discuss how regulators could accelerate safe access to these therapies.

In September, the NIH announced the Building Evidence and Collaboration for Genomics in Nationwide Newborn Screening (BEACONS) initiative to explore integrating whole-genome sequencing into newborn screening programs, focusing on conditions treatable in the first year of life. At the same time, the NIH’s Somatic Cell Genome Editing (SCGE) program is advancing the science of precisely editing DNA within the cells of living patients.

Most conservatives are wary of gene editing, and for good reason. The term covers two very different practices with profoundly different consequences.

Germline gene editing, for example, alters DNA in sperm, eggs, or embryos, which means the changes are inherited by every cell in the resulting child and passed down to future generations. Many scientists and ethicists oppose this approach because unintended edits, known as off-target effects, could introduce new problems. Such gene editing is like changing a book’s “master template” in a printing press such that every copy produced is permanently different.

In contrast, somatic gene editing alters DNA only in specific cells or tissues of a born person, such as in blood cells to treat sickle-cell disease. These changes are not heritable and affect only that individual, like repairing a single copy of a book already in circulation. This approach is responsible for the innovative research being done to save lives. Such treatments prioritize root cause care within a person’s body without destroying or harming human embryos.

Baby KJ and New Horizons in Gene Editing

Since SCGE launched in 2018, the NIH has prioritized innovative genome editing tools that target areas “that are harder to reach such as the brain, ear, heart, and lung,” according to the SCGE report summary. Researchers designed gene-editing tools, known as “prime editors,” that can correct nearly 90 percent of known disease-causing variants.

The power of the SCGE program’s research was demonstrated earlier this year in the case of Baby KJ. His miraculous recovery followed the first personalized gene-editing treatment ever given to an infant with a fatal condition.

Baby KJ was diagnosed shortly after birth with CPS1 deficiency, one of the deadliest urea cycle disorders. Without immediate intervention, toxic ammonia rapidly builds up in the blood, triggering seizures, coma, and death. Historically, more than half of affected newborns die in infancy, and even with the most aggressive treatment, many suffer severe neurological damage or must undergo a liver transplant simply to survive.

But for Baby KJ, that grim prognosis no longer had to be his fate. Instead of facing a lifetime of invasive treatments and the looming threat of early death, he received an innovative and life-affirming somatic gene-editing therapy designed to correct the mutation in his liver cells. This groundbreaking intervention reversed the fatal course of his disease and offered him the chance of a normal, healthy life.

This case, according to NIH scientist Joni Rutter, “promises a new era of precision medicine for hundreds of rare diseases.” If such treatments can effectively offer root cause treatments for these rare genetic conditions, imagine the possibilities for more common diseases.

Screening Is Not Healing

While researchers with the NIH and biotech labs labor to develop therapies that treat and cure disease, a parallel industry is moving in a radically different direction. Companies like Orchid promise parents the ability to optimize their future children before they are even born. Their embryo screening services offer reports on more than 1,200 single-gene disorders, dozens of polygenic conditions, sex, and even the potential to screen for non-disease traits such as eye color, intelligence, and personality. Noor Siddiqui, Orchid’s founder, told author and New York Times columnist Ross Douthat that this approach is “more affordable” than pharmaceutical treatments once the child is born.

It’s easy to see why this framing is so appealing: why wait for complex cures when you can simply select a healthy human embryo? But the process hides something darker. Embryonic genetic screening does nothing to cure disease; it merely offers reports about which human embryos may carry unwanted diseases or traits. The implication, of course, is that unwanted embryos should be destroyed in favor of the healthiest, smartest, and “best” child.

Even more troubling, this kind of technology undermines the motivation to create real treatments like the one that saved Baby KJ’s life. If it is more profitable to screen out blindness or the risk of cancer at the embryonic stage, why invest in the painstaking research needed to treat blindness or cure cancer in living patients?

Despite their promise, both preimplantation genetic testing for aneuploidy (PGT-A) and whole-genome sequencing (WGS) suffer from significant accuracy problems. Multiple studies show that PGT-A often misclassifies embryos: one reanalysis found that 33 percent of embryos labeled “abnormal” were actually normal, while another revealed a false-positive rate of nearly 55 percent due to mosaicism, where embryos naturally contain a mix of normal and abnormal cells. This mosaicism can also allow embryos to self-correct, making early genetic assessments unreliable.

Other research shows that PGT-A has no proven benefit for increasing live birth rates and, in some cases, it may even lower them. WGS faces similar challenges. Because it relies on amplifying tiny amounts of DNA from a few embryonic cells, results are often unclear and are inherently probabilistic. As one study put it, WGS for embryo selection “is not advisable” due to “analytical and clinical limitations.”

Professional bodies have echoed these cautions. In 2024, the American College of Medical Genetics and Genomics (ACMG) argued that polygenic embryo screening “should not currently be offered as a clinical service.” As they argue, “The implementation of PGT-P has been challenged by several groups of scientists and professional societies, including the American Society of Human Genetics, the European Society of Human Genetics, and the European Society of Human Reproduction and Embryology, all of which have called the utilization of PGT-P unethical and reject its use in clinical care.”

Even if embryo screening worked perfectly, it would still raise significant moral and ethical concerns.

Siddiqui herself acknowledged that “any embryo testing—any testing on embryos, period—is still a screening test. Until that baby is actually born, you can’t give a definitive diagnosis.” Yet companies continue to market these tests as reliable predictors of a child’s future health and traits.

Even if embryo screening worked perfectly, it would still raise significant moral and ethical concerns. It reduces human life to a list of potential traits, such as a person’s health, sex, IQ, personality, or appearance. This is consumer eugenics: a belief that we can design better people by rejecting the human embryos who don’t appear to measure up.

The contrast with somatic gene and cell therapy could not be sharper. As journalist Ari Schulman puts it, “Cancer screening prevents disease by helping the patient live. Embryo screening prevents disease by killing the patient.” In one case, scientists harness somatic gene editing to heal and restore the human person. In the other, scientists use embryonic genetic “screening” to filter and discard the human embryos. As Schulman notes, embryonic genetic screening neither cures nor treats disease, nor does it alter or improve the traits of a given child; it merely selects which potential lives are permitted to continue. Somatic gene editing, by contrast, represents an admirable scientific advance that seeks to treat genetic disease in order to save or improve the lives of men and women—without resorting to the selection or destruction of human life.

If the goal of medicine is to protect and restore life, then our efforts and investments should flow to therapies that treat the sick, not to technologies that eliminate them before they are born. It’s time to reject the false promise of embryo screening and instead pursue treatments that are innovative, restorative, and life-affirming.