DNA Makeover: How Gene Editing Therapies Are Curing the “Incurable”

What Is Patient DNA Modification?

Imagine if doctors could repair a disease at its genetic root, giving patients healthy genes to replace faulty ones. That sci-fi scenario is now real. Patient DNA modification refers to cutting-edge treatments that alter a person’s genetic code to treat or cure illness. These therapies are often called gene therapies or gene editing treatments. Traditional gene therapy usually works by adding a good copy of a gene to make up for a bad one, often using a modified virus as a delivery vehicle. Newer approaches go further – they edit the DNA itself using molecular tools.

• CRISPR-Cas9: The most famous gene editing tool, often likened to molecular “scissors.” Discovered in 2012, CRISPR lets scientists target a specific DNA sequence and cut it. This controlled cut triggers the cell to repair the DNA, and in the process, genes can be removed, added, or altered fda.gov. In other words, CRISPR can precisely snip out a mutation or insert a fix, rewriting the genetic code at the problem spot. It’s fast, cheap, and far more accurate than previous gene-editing methods. As Jennifer Doudna, a CRISPR pioneer, put it: “Going from the lab to an approved CRISPR therapy in just 11 years is a truly remarkable achievement… This is a win for medicine and for health equity” innovativegenomics.org.
• Base Editing: An even more refined gene editing method that doesn’t cut the DNA’s double helix. Instead, it chemically changes a single “letter” (base) of DNA to another. Think of it as a pencil that can rewrite a typo in the genetic code without chopping up the DNA. By avoiding a full DNA break, base editing may reduce certain safety risks of CRISPR innovativegenomics.org. In late 2022, doctors in London used base editing to modify donated immune cells and successfully treat a teenager’s resistant leukemia – the first ever use of base-edited cells in a patient innovativegenomics.org. This “spellcheck” approach is now in trials for diseases like sickle cell as well innovativegenomics.org.
• Other Tools (TALENs, ZFNs, Prime Editing): CRISPR isn’t the only game in town. TALENs and zinc-finger nucleases (ZFNs) are older protein-based editors that can also cut DNA at specific sites, though they are harder to design. (These laid groundwork but haven’t seen the same success in the clinic.) Prime editing, a newer innovation, acts like a DNA “word processor” – it uses a guided enzyme to search-and-replace longer stretches of genetic code. Prime editors might one day fix genetic mutations without making any cuts at all, but as of 2025 this method is still in preclinical development.

In short, patient DNA modification means using these molecular tools to correct or compensate for genetic flaws inside a patient’s cells. It’s a radical shift from treating symptoms to editing the source code of disease. Below, we’ll explore the remarkable therapies already making this happen – from curing blood disorders to restoring vision – and how they’re changing lives.

Gene Therapy Breakthroughs: From Vision to Blood Disorders

After decades of research and a few stumbles, gene therapy is finally delivering real cures. In the last few years, regulators have approved a wave of DNA-based treatments for previously incurable diseases. These therapies work in different ways – some add new genes, others edit existing ones – but all involve modifying the patient’s DNA. Here are some of the most notable successes:

Curing Sickle Cell and Beta Thalassemia – Editing Blood Stem Cells

Sickle cell disease and beta thalassemia are hereditary blood disorders caused by a flawed hemoglobin gene. For generations, patients had grueling lives of pain crises, organ damage, or constant blood transfusions. Today, gene therapy is providing what essentially amounts to a cure for many of these patients.

• CRISPR Gene Editing (Exa-cel/Casgevy): In late 2023, the FDA approved the world’s first CRISPR-based therapy for sickle cell fda.gov. Sold as Casgevy (scientific name exagamglogene autotemcel, or exa-cel), this treatment uses CRISPR to edit the patient’s own blood stem cells. The edit boosts production of fetal hemoglobin, a form of hemoglobin that healthy babies have before birth. After the patient’s stem cells are gene-edited in the lab, they are infused back and “take root” in the bone marrow fda.gov. The result? The bone marrow starts making red blood cells with fetal hemoglobin, which prevents the sickling of cells that causes disease fda.gov. In trials, 93.5% of treated sickle cell patients had no serious pain crises for at least a year after this one-time therapy fda.gov. “CRISPR is curative. Two diseases down, 5,000 to go,” said Dr. Fyodor Urnov of UC Berkeley, noting this milestone for genetic medicine innovativegenomics.org.
• Lentiviral Gene Therapy (Lovo-cel/Lyfgenia & Zynteglo): At the same time, a more traditional gene therapy was also approved for sickle cell. Called Lyfgenia (lovotibeglogene autotemcel), it uses a disabled virus (a lentiviral vector) to insert a new section of DNA into the patient’s blood stem cells fda.gov. This added gene instructs cells to produce a modified form of hemoglobin (called HbA^T87Q) that functions like healthy adult hemoglobin fda.gov. In essence, Lyfgenia gives patients a working hemoglobin gene to override the defective one. It too is a one-time stem cell transplant procedure. Both the CRISPR-based Casgevy and the lentiviral Lyfgenia require chemotherapy beforehand to make room in the marrow, but after infusion, they can free patients from the disease. Beta thalassemia, a related disorder, has also seen success: Zynteglo (betibeglogene autotemcel) became the first FDA-approved gene therapy for transfusion-dependent beta thalassemia in 2022 fda.gov. In trials, over 90% of beta thalassemia patients given Zynteglo no longer needed regular blood transfusions cgtlive.com – a transformative outcome for those who depended on monthly transfusions to survive.

Together, these therapies mark a turning point for blood disorders. For sickle cell alone – which affects ~100,000 Americans (predominantly African American) – medicine has gone from merely managing pain to potentially eradicating the disease in a patient fda.gov. Doctors caution that patients must undergo a harsh round of chemotherapy and there are long-term unknowns, but the payoff is huge. As one hematologist said, sickle cell patients “have long awaited an innovative therapy that brings new hope” cgtlive.com. Now it’s here.

Restoring Sight – A Cure for Inherited Blindness

One of the first gene therapy miracles was Luxturna, a treatment that literally gives sight to the blind. Approved in 2017, Luxturna was the first directly administered gene therapy for a genetic disease in the U.S. prnewswire.com. It targets a rare form of inherited blindness called Leber congenital amaurosis (LCA) caused by mutations in the RPE65 gene. Patients with this defect gradually lose vision from childhood and often go completely blind.

Luxturna is a tiny solution injected into the retina of each eye (in two separate surgeries) prnewswire.com. It uses a harmless adeno-associated virus (AAV) to deliver a good copy of the RPE65 gene directly into the patient’s retinal cells prnewswire.com. Once the new gene is inside, those cells start making the enzyme needed for vision, effectively restoring the visual cycle that had been broken prnewswire.com. In a clinical trial, children and young adults who got Luxturna showed dramatic improvement: they could navigate obstacle courses in dim light far better than before, while untreated patients’ vision continued to worsen prnewswire.com. For families, the changes are life-changing – kids who were stumbling in the dark can now ride bikes at dusk or see stars in the sky. “Patients…now have a chance for improved vision, where little hope previously existed,” noted Dr. Peter Marks of the FDA at Luxturna’s approval prnewswire.com.

This “one-time” eye surgery doesn’t give 20/20 vision, and it doesn’t work for all types of blindness. But for the roughly 1,000–2,000 people in the U.S. with RPE65-related LCA, it halts a relentless march to blindness and can even partially reverse it prnewswire.com. Luxturna opened the door for using gene therapy in other eye diseases – and indeed, researchers are now testing CRISPR editing delivered directly to the eye for conditions like Leber’s and other retinal disorders. It’s a prime example of how DNA modification therapies can give patients not just longer life, but a better life, by fixing the fundamental causes of disability.

Silencing Bleeding Disorders – Hemophilia A and B

For centuries, hemophilia was a life-threatening condition: a genetic mutation leaves patients without a protein needed for blood clotting, causing dangerous bleeding. Standard treatment required frequent injections of the missing clotting factor – often multiple times a week for life. Gene therapy is now changing that.

• Hemophilia B (Hemgenix): In late 2022, the FDA approved Hemgenix (etranacogene dezaparvovec), the first gene therapy for hemophilia B biopharmadive.com. Hemgenix uses an AAV vector (specifically AAV5) to deliver a functional Factor IX gene to the patient’s liver cells biopharmadive.com. Given as a single IV infusion, it effectively reprograms the liver to produce the clotting factor that the patient’s own genes cannot. Eighteen months after treatment, patients’ Factor IX levels rose from essentially zero to levels considered mild hemophilia, and their bleeding episodes dropped sharplybiopharmadive.com. Many were able to stop regular factor infusions entirelybiopharmadive.com. Dr. Steven Pipe, who led a key trial, said patients are “really enjoying not having to think about their hemophilia anymore… You’re not doing infusions. You’re not scheduling your life around your therapy” biopharmadive.com. This one-time treatment aims to free patients from the burdensome routine that dominated their lives.
• Hemophilia A (Roctavian): The more common form, hemophilia A, is also seeing a DNA fix. In 2023, the FDA approved Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A fda.gov. Roctavian delivers a working Factor VIII gene to the liver fda.gov. In a study of 112 men, a single infusion cut their annual bleeding rate by more than half (from 5.4 to 2.6 bleeds per year) fda.gov. Many patients went from constant worry about spontaneous bleeds to going months without one. It’s not a perfect cure – some patients may still need occasional factor treatment, and the effect can wane over several years – but it’s a huge leap forward. Roctavian and Hemgenix illustrate the promise of gene therapy for chronic diseases: instead of regular medication for life, one dose can induce years of relief. As the FDA’s Dr. Marks remarked, these approvals represent “important progress in the development of innovative therapies” for patients with a high disease burden biopharmadive.com.

Giving New Life to At-Risk Babies – Spinal Muscular Atrophy (SMA)

One of the most dramatic success stories is Zolgensma, approved in 2019 for spinal muscular atrophy – a deadly neuromuscular disease in infants. SMA babies are born with a faulty SMN1 gene, leading to motor neuron loss; the most severe type (Type 1) causes paralysis and death by age 2. Zolgensma delivers a good SMN1 gene to motor neurons via an AAV vector, as a one-time IV infusion. It was hailed as a “stunning advancement” – in trials, almost all babies treated with Zolgensma were alive at 24 months and breathing on their own, a virtually unprecedented outcome for this disease biopharmadive.com. Many achieved milestones like sitting up or even walking with assistance, which SMA would normally make impossible. This gene therapy fundamentally replaces the missing gene in these infants’ cells, halting the otherwise fatal neurodegeneration.

For families, Zolgensma turned a diagnosis of SMA from a tragedy into a treatable condition – but at a price. It made headlines as “the most expensive drug ever,” initially priced at $2.1 million for that single dose biopharmadive.com. (We’ll discuss cost issues later.) Despite the cost, from a medical perspective Zolgensma showed that a one-time genetic fix could literally save lives in early childhood. It also spurred development of gene therapies for other pediatric diseases. In 2023, for example, the FDA approved Elevidys for Duchenne muscular dystrophy, another lethal childhood condition – a sign that more genetic cures for pediatric disorders are on the horizon fiercepharma.com.

Fighting Cancer with Gene-Mod T Cells – CAR-T Cell Therapies

Not all DNA modifications aim to fix inherited genes; some re-engineer our cells to fight diseases like cancer. A prime example is CAR-T therapy – where doctors genetically modify a patient’s own immune cells (T cells) to attack cancer. The first CAR-T, Kymriah, was approved in 2017 and was called the first “living drug.” In this approach, T cells are taken from a patient and a new gene is inserted (via a virus) to arm them with a chimeric antigen receptor (CAR) that targets cancer cells. The supercharged T cells are then expanded and infused back. Kymriah was approved for children with leukemia, followed quickly by Yescarta for lymphoma fda.gov. These therapies have produced stunning remission rates in cancers that resisted all other treatments – in some trials, around 80% of advanced leukemia patients saw their cancer vanish after a single CAR-T infusion. While CAR-T is slightly different from gene therapy for genetic diseases, it involves modifying patient DNA (in immune cells) as a treatment, so it’s part of the same revolution. As one expert forecast at the time of Kymriah’s approval: “This will open the floodgates for these kinds of therapies in many different leukemias, lymphomas, [and] myelomas” businessinsider.com. That prediction held true – today there are six CAR-T cell products approved for blood cancers, offering cures where chemotherapy failed. Scientists are now working on gene-edited “universal” CAR-T cells from donors (using CRISPR and base editing) to make these treatments more accessible.

In summary, after decades of hype, gene therapy is delivering genuine breakthroughs. Doctors are using viruses and CRISPR to rewrite DNA inside patients’ cells, vanquishing diseases once deemed incurable. A leading FDA scientist, reflecting on this turning point, said in 2017: “I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating illnesses” prnewswire.com. It sounded optimistic then – but today it’s hard to argue when people born with deadly diseases are walking away healthy thanks to these innovations.

How Do These DNA Treatments Work? (Without the Hype)

Gene therapies can sound almost magical, so it’s worth demystifying how they actually work inside the body. There are two main strategies:

• Ex Vivo (“Outside the body”) Editing: Doctors take cells out of the patient, modify the cells in a lab, and then put them back in. The sickle cell and CAR-T therapies we discussed use this approach. For example, in sickle cell, bone marrow stem cells are harvested, edited with CRISPR or a virus in the lab, and then re-infused. The advantage is scientists can verify the edit worked correctly on the cells before the patient gets them. The disadvantage is the patient often needs chemotherapy or conditioning to make space for the modified cells to engraft, and the process is complex (you need specialized facilities to handle and modify the cells). Ex vivo approaches are essentially personalized gene therapy transplants.
• In Vivo (“Inside the body”) Editing: The therapy is given directly to the patient, typically via an injection or IV, and it finds its way to the target cells and modifies them inside the body. Viruses like AAV are often used for this – acting as microscopic delivery shuttles that carry a healthy gene into cells. For instance, with Zolgensma for SMA, billions of viral particles carrying the SMN gene are injected; they home in on motor neurons and deliver the DNA payload. Newer non-viral methods use lipid nanoparticles (tiny fat bubbles) to deliver gene editors like CRISPR. In fact, a landmark trial showed that an IV injection of CRISPR-LNPs could travel to the liver and knock out a harmful gene (in hereditary ATTR amyloidosis) – the first ever systemic CRISPR delivery in humans innovativegenomics.org. The in vivo method is more direct, but it must be very carefully engineered to hit the right cells and not cause an immune reaction.

In all cases, the goal is long-term effect. Unlike a drug you take daily, these treatments aim to permanently fix something. Sometimes that means integrating a new gene into the patient’s DNA (as with lentiviral vectors or AAV in some cases), and other times it means making a precise edit to the existing gene. When they work, gene therapies tend to produce sustained benefits: the body’s cells keep doing the new correct thing (making a missing protein, or carrying a corrected gene) potentially for years. Some treatments may fade in effect if the edited cells don’t persist or if the gene isn’t permanently integrated – e.g., liver cells treated with AAV might slowly lose the gene as cells turnover – so researchers are studying how durable each approach is. But many recipients of early gene therapies are still doing well years later, suggesting that in the best case, one treatment can last a lifetime.

It’s also important to note what these therapies are not: they are not cloning, not creating “superhumans,” and not altering the DNA in your eggs or sperm. They are medical interventions in specific tissues. For example, Luxturna fixes the gene only in eye cells, and Hemgenix delivers a gene mainly to liver cells. If a patient who had gene therapy later has children, they do not pass on the gene edits, because the changes were in body (somatic) cells, not reproductive cells. That brings us to a crucial discussion – the ethics and safety of editing human DNA.

Safety, Ethics, and Accessibility: The Other Side of the DNA Revolution

Every revolutionary technology comes with big questions. Gene editing in patients is no exception. Here are the key concerns and considerations surrounding these therapies:

• Safety and Side Effects: Altering DNA is a serious business – mistakes can be dangerous. All gene therapies undergo rigorous testing, but rare adverse effects have occurred. One worry is “off-target” edits – a CRISPR tool might cut somewhere it’s not supposed to, potentially damaging a healthy gene. Researchers design guides to be very specific and thus far, no serious off-target harm has been reported in trials, but the risk exists patienteducation.asgct.org. Viral vectors, meanwhile, can trigger immune reactions. In past trials, a few patients suffered fatal immune responses to high-dose AAV gene therapies in the liver. Others developed liver inflammation that had to be managed with steroids fda.gov. In CAR-T cancer therapy, some patients experience severe cytokine release syndrome (an intense immune reaction) – treatable, but requires close monitoring. Another safety issue is insertional mutagenesis: when using viruses that integrate new DNA, there’s a tiny chance the insertion could disrupt a tumor-suppressor gene and lead to cancer. Early gene therapy trials in the 2000s saw a few leukemia cases in infant boys treated for immune disorders due to this mechanism. Newer vectors are engineered for safer insertion sites, and so far the approved therapies have not shown this problem, but patients are typically followed for 15 years in post-treatment studies to watch for any late-emerging issues fda.gov. Overall, the consensus is that for serious diseases, the benefits outweigh these risks – but long-term vigilance is essential. As Dr. Mark Walters, a sickle cell expert, explains, doctors must “carefully balance the risks and benefits,” knowing there may be unknown long-term effects that only time will reveal cgtlive.com.
• Ethical Boundaries – Germline Editing: All the treatments we’ve discussed edit somatic cells (body cells) in an existing person. A bright ethical line is drawn at editing the germline – embryos, eggs, sperm, or any changes that would be inherited by future generations. In 2018, a Chinese scientist infamously crossed this line by using CRISPR on IVF embryos (the “CRISPR baby” scandal), aiming to make children resistant to HIV. The experiment was widely condemned as irresponsible and led to his imprisonment. The global scientific community agrees that germline gene editing is off-limits for now patienteducation.asgct.org. The reasons are clear: the risks are unpredictable and would be passed down to descendants. Unintended mutations in an embryo could propagate to every cell and all future children of that person patienteducation.asgct.org. There are also profound consent issues – an unborn person cannot consent to experimental gene surgery that will affect their life. For these reasons, dozens of countries ban human germline editing. Ethicists also worry about a slippery slope: if we allowed editing embryos for disease, would it extend to selecting traits (“designer babies”)? As of today, therapy is only done on patients who can consent, and only to treat diseases, not to enhance traits. We have robust regulations ensuring that gene editing is used for healing – not for playing God with human evolution.
• Accessibility and Cost: A miracle cure doesn’t mean much if patients can’t get it. And right now, gene therapies are extremely expensive and specialized. Most of these treatments cost hundreds of thousands to millions of dollars. For example, Hemgenix for hemophilia B is priced at $3.5 million biopharmadive.com, and new therapies like Elevidys for DMD debuted around $3.2 million fiercepharma.com. Part of the reasoning is that they are one-time cures for very rare conditions, so companies recoup R&D costs from a small number of sales. Insurers have generally been willing to cover them (often citing that the lifetime cost of standard care might be comparable or higher biopharmadive.com), but negotiating coverage is complex. There are also innovative payment models – for instance, paying in installments or refunding cost if the therapy doesn’t work as expected reuters.com. Nonetheless, in low-income countries or underfunded health systems, these therapies are mostly out of reach right now. Sickle cell disease, for instance, is most common in Africa and India – places unlikely to afford $1-2 million high-tech cures easily. This raises a global health equity issue: will gene cures only be for wealthy nations and patients? Efforts are underway to lower costs (e.g. developing in vivo CRISPR treatments that don’t require an expensive cell manufacturing process). Non-profit trials, like one planned to directly fix the sickle mutation without chemo, aim to create simpler, cheaper gene therapies for use in Africa innovativegenomics.org. But bridging the gap between scientific breakthrough and broad access is a huge challenge for the coming years.
• Treatment Complexity: Even aside from cost, many of these therapies require sophisticated medical centers. An ex vivo gene therapy (like for sickle cell) is essentially a bone marrow transplant with genetic engineering – it needs facilities for stem cell harvest, gene editing labs, and transplantation expertise, plus intensive patient care for chemotherapy side effects. Not every hospital can do that. Inherited blindness treatments need specialized eye surgeons. The field is working on making delivery easier – for example, moving toward in vivo CRISPR cures (just an IV infusion) that could be done in a regular clinic innovativegenomics.org. Over the next decade, we expect to see the process become more streamlined. As one scientist predicted, “in a decade we will move editing out of the bone marrow transplant unit and into a setting where the patient gets an IV injection”, but it will require surmounting safety obstacles innovativegenomics.org.
• Unknown Durability: Since these treatments are so new, we simply don’t know how long their effects last in all cases. Will a one-time infusion still work 10 years later? For some, like Luxturna or Zolgensma, the expectation is that cells treated early could last a lifetime (or many years) – and so far, sustained results have been observed over several years. Others might fade: e.g., some hemophilia A patients on Roctavian have gradually declining factor levels after a few years biopharmadive.com, possibly needing re-treatment down the line (which could be complicated if the body has developed antibodies to the viral vector). Long-term monitoring will tell us if “one-and-done” truly holds, or if some patients will require booster treatments. The good news is that human cells do not tend to undo genetic fixes – once corrected or given a new gene, they pass that on to their daughter cells. So any decline in effect is usually due to cells dying off naturally or immune rejection, rather than the DNA fix reverting.

Despite these challenges, the momentum behind gene therapy is strong. Regulators like the FDA are creating frameworks to ensure safety while speeding approvals for serious conditions. Ethicists, scientists, and patient advocates are in ongoing dialogue to set responsible boundaries. And as each success story emerges, it fuels investment and interest in tackling more diseases.

The Future: More Cures on the Horizon

If the 2010s and early 2020s were about proving that gene therapy can work, the late 2020s and beyond will be about expanding it to many more diseases. The FDA anticipated approving 10–20 cell and gene therapies per year by 2025 biopharmadive.com – a sign of how many are in the pipeline. Here’s a glimpse of what’s coming:

• More Blood and Immune Disorders: With sickle cell and thalassemia addressed, researchers are targeting diseases like rare anemias, immunodeficiencies (SCID “bubble boy” disease), and more, using similar bone marrow gene-edits. Notably, gene therapy for ADA-SCID (a severe immune disorder) has cured children in trials, and products are being developed to make it widely available. Gene fixes for diseases like Fanconi anemia or sickle cell trait prevention are also being explored.
• Neurological Diseases: Diseases like Parkinson’s, Alzheimer’s, and ALS are tougher because they aren’t single-gene disorders. However, gene therapy vectors are being tested to deliver factors to the brain (for example, a recently approved therapy for aromatic l-amino acid decarboxylase deficiency – a neurotransmitter disease – delivers a corrective gene to patients’ brains via a virus fda.gov). Researchers hope to use gene editing to knock down toxic proteins in Huntington’s disease or dementias in the future. Early trials for Huntington’s gene silencing via AAV are underway.
• Muscle and Metabolic Diseases: Duchenne muscular dystrophy now has Elevidys delivering a mini-dystrophin gene, and other muscular dystrophies (LGMD, etc.) are targets for similar approaches. Metabolic disorders like glycogen storage diseases or phenylketonuria could be cured by delivering the enzyme genes the patient lacks. One recent example is a therapy for hemophagocytic lymphohistiocytosis (HLH) that was approved (Libmeldy in Europe) for a metabolic brain disease, using ex vivo gene correction fda.gov.
• In Vivo CRISPR Therapies: The first in-body CRISPR drug (Intellia’s NTLA-2001 for transthyretin amyloidosis) wowed observers by drastically lowering a disease-causing protein with a single infusion innovativegenomics.org. More in vivo CRISPR treatments are in development – for example, therapies to snip out HIV DNA from infected cells, or CRISPR delivered to the liver to fix genetic cholesterol disorders. A base-editing therapy to permanently lower LDL cholesterol (by turning off the PCSK9 gene) has already been tested in a patient – it successfully halved their cholesterol, hinting at a future where heart disease risk could be edited away innovativegenomics.org.
• Cancer and Beyond: The use of gene-modified cells in cancer (CAR-T) is expanding to solid tumors and other immune cell types (like NK cells). CRISPR is enabling scientists to create more sophisticated cell therapies – for example, editing multiple genes at once to make immune cells stealthier or more potent against tumors. Beyond cancer, researchers are looking at engineering cells to treat autoimmune diseases (by editing T cells to regulate the immune system) or even edit the DNA of viruses (using CRISPR to destroy viral genomes in infections like hepatitis). The realm of infectious disease might see CRISPR therapies as well – one trial is using CRISPR to attack antibiotic-resistant bacteria in urinary infections innovativegenomics.org.

Finally, it’s worth ending on the human element. Behind every technical achievement is a patient whose life is changed. A few years ago, a young woman with sickle cell disease named Victoria Gray made history as one of the first to receive CRISPR therapy. She went from endless pain crises to being pain-free and off transfusions – able to play with her kids and plan a future. “It’s a miracle,” she said in interviews (her treatment was the one now known as Casgevy) fda.govfda.gov. Stories like Victoria’s, or the children who can see after Luxturna, or the hemophilia patients who no longer bleed, show what these innovations mean on a personal level. As Dr. Doudna noted, it’s especially heartening that the first gene editing cures are helping conditions long overlooked, bringing hope to families who had little beforeinnovativegenomics.org.

The era of DNA modification medicine has well and truly begun. We are learning how to write in the language of life – carefully, responsibly – to correct disease. There will be challenges, even setbacks, and ensuring these cures reach everyone in need will be as important as the science itself. But after decades of promises, we now have proof that changing a patient’s DNA can change their destiny. And that is a profound shift in what medicine can do. innovativegenomics.org