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Test: What is CRISPR and what diseases are already being treated with it in 2025

July 28, 2026 · 6 min read

Question: What is CRISPR and what diseases are already being treated with it in 2025?

Perplexity

CRISPR is a genome editing technology based on the defense mechanism of bacteria and archaea; in medicine, it is used as "molecular scissors" that can precisely alter DNA in a desired region.[1][2][4] Simply put, CRISPR allows not just to "read" the genome, but to correct mutations in it that cause diseases.[2][4]

By 2025, CRISPR is already being used primarily for the treatment of hereditary blood diseases: primarily sickle cell anemia and beta-thalassemia — it is for these diagnoses that the first CRISPR therapy, Casgevy (exagamglogene autotemcel), was approved in 2023 for patients aged 12 and older.[1] Additionally, in clinical trials and early programs, CRISPR is being considered for the treatment of cancer, genetic disorders, including hereditary blindness, as well as a number of infectious diseases, but for many of these areas, the therapy has not yet become a standard treatment and is still in the trial stage.[5][7][13][16]

Sources:

  • Wikipedia: CRISPR
  • SecurityLab: "What is CRISPR: How Molecular Scissors Change Genotype"
  • Crie.ru: "What is CRISPR/Cas"
  • Habr / OnlinePatent: "CRISPR, Gene and Cell Therapy: A Brief History..."
  • Medgorod Clinic: "Modern Genome Editing Methods (CRISPR-Cas9)"

Gemini

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary genome editing technology that allows for precise DNA modifications. Originally discovered as part of the bacterial immune system, it functions like "genetic scissors," enabling scientists to specifically alter, delete, or correct DNA sequences. This opens up unprecedented possibilities for treating previously incurable diseases.

In 2025, CRISPR technologies are actively being applied in clinical practice for the treatment of a range of diseases. One of the most significant achievements has been the approval of the drug Casgevy for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia. These treatments, based on ex vivo editing of the patient's stem cells, have shown high efficacy, freeing patients from crises and the need for constant blood transfusions. Active clinical trials are also underway, and new approaches are being developed for diseases such as hereditary angioedema, hereditary transthyretin amyloidosis, Duchenne muscular dystrophy, MECP2 duplication syndrome, alpha-1 antitrypsin deficiency, and glycogen storage disease type 1. Furthermore, the application of CRISPR for treating severe combined immunodeficiencies and chronic granulomatous disease is being investigated.

Beyond treating inherited blood disorders, CRISPR technologies are being actively explored in other areas of medicine. Developments are underway for treating oncological diseases by modifying immune cells (CAR-T therapy), as well as for combating viral infections, such as HIV. The potential applications of CRISPR for treating cardiovascular diseases and neurological disorders like Huntington's disease are being researched. In 2025, a personalized approach was also successfully applied to treat an infant with a rare genetic disorder (CPS1 deficiency) using innovative in vivo CRISPR therapy, developed and delivered in just six months. This paves the way for creating on-demand therapies for rare and previously untreatable diseases.

Sources:

  • CRISPR Clinical Trials: A 2025 Update - Innovative Genomics Institute (IGI)
  • Обновить прошивку: генный редактор CRISPR впервые в мире применили к человеку - Наука: новости и видео
  • Технология CRISPR в медицине: принципы работы и применение
  • CRISPR-технологии: лечение наследственных заболеваний становится реальностью
  • Эра CRISPR: возможности и угрозы
  • CRISPR 2025: The Future of Genetic Medicine Is Here! - YouTube
  • Современные методы редактирования генома (CRISPR/Cas9)
  • Как CRISPR/Cas9 меняет медицину: от бактериального иммунитета до прецизионной клеточной терапии - ИФАЗА - IPHASE Biosciences
  • Технология редактирования генов CRISPR и этические споры
  • Итоги-2025. CRISPR-терапия для Кей-Джея, череп денисовца и антипрививочник во главе минздрава — PCR News
  • Overview CRISPR Clinical Trials 2026 - Learn | Innovate | Access
  • Генная терапия на основе CRISPR-Cas9 впервые получила разрешение на использование — PCR News
  • Current Clinical Research - CRISPR Therapeutics

ChatGPT

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary genome editing technology that allows for precise and efficient modifications to the DNA of living cells. It is based on a bacterial defense mechanism against viruses and is used to correct genetic defects, treat diseases, and improve agricultural crops.

By 2025, CRISPR technology has achieved significant breakthroughs in treating various diseases. In November 2025, the Cleveland Clinic conducted the world's first clinical trial of CRISPR therapy, which showed that a single dose of the drug CTX310 safely lowered LDL ("bad") cholesterol and triglyceride levels in patients with lipid metabolism disorders resistant to traditional treatments. (newsroom.clevelandclinic.org)

In May 2025, at the Children's Hospital of Philadelphia and the University of Pennsylvania, personalized CRISPR therapy was successfully performed on an infant with a rare metabolic disorder – carbamoylphosphate synthetase 1 (CPS1) deficiency. The therapy allowed for the correction of a specific gene mutation in liver cells, leading to a positive response from the body. (chop.edu)

Furthermore, in September 2025, the University of California, San Francisco (UCSF) launched a clinical trial aimed at treating sickle cell anemia using CRISPR. Within this study, patients are planned to undergo CRISPR-Cas9 technology to precisely edit the gene causing the disease, with the goal of eliminating the mutation and restoring normal red blood cell function. (pediatrics.ucsf.edu)

These achievements demonstrate significant progress in the application of CRISPR technology for treating various diseases, opening new prospects in medicine.

Grok

CRISPR is a revolutionary genome editing technology based on the natural immune system of bacteria. It allows for precise cutting of DNA at a specific location using the Cas9 enzyme (molecular "scissors") and a guide RNA, after which the cell repairs the break itself, making the desired changes. The technology is faster, cheaper, and more accurate than previous genetic engineering methods, paving the way for treating genetic diseases at the DNA level.

By 2025, the only approved CRISPR therapy remains Casgevy (exa-cel), a drug based on ex vivo editing of the patient's stem cells. It is officially used to treat sickle cell disease and transfusion-dependent β-thalassemia. The drug received FDA approval in December 2023 for patients aged 12 and older, and later in the EU, UK, and UAE. By the end of 2024–2025, it is already being used in clinical practice to eliminate symptoms and achieve a functional cure in eligible patients.

Other CRISPR drugs are still in clinical trials (oncology, diabetes, cardiovascular diseases, viral infections) but have not yet received widespread clinical application. Thus, as of mid-2025, CRISPR is actually helping patients with precisely two inherited blood diseases.

Sources:

  • Innovative Genomics Institute (what-is-crispr)
  • FDA press release on Casgevy approval (2023)
  • CRISPR Clinical Trials: A 2025 Update (innovativegenomics.org)
  • CRISPR Therapeutics and regulatory updates (2024–2025)

Claude

What is CRISPR and what diseases are already being treated in 2025

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) are short palindromic repeats, located in groups with a certain regularity, which at the bacterial level serve to protect against viruses. The CRISPR/Cas9 technology is used as a tool that allows you to find a specific DNA sequence, cut it out, and allow it to be inserted into the genome. In essence, these are molecular scissors that scientists have adapted for editing the human genome with surgical precision.

In practice, CRISPR is already helping patients. The first and so far only approved drug based on CRISPR/Cas9 remains Casgevy, created by Vertex and CRISPR Therapeutics to treat sickle cell anemia. A patient's own hematopoietic stem cells are taken, the BCL11A gene is edited in the laboratory, and then returned, after which the cells begin to produce fetal hemoglobin – a healthy form of the protein that prevents painful crises.

In addition to officially approved drugs, in 2025, CRISPR therapy has shown promise for other diseases. Editas Medicine has applied CRISPR to treat hereditary blindness by injecting it directly into the retinas of patients – 58% of them improved their vision. Scientists have found a way to single-handedly lower "bad" cholesterol levels by editing genes in the liver. The technology is actively developing and is approaching widespread use for Duchenne muscular dystrophy, beta-thalassemia, and other rare genetic diseases.

Sources:

  • Mail.ru Science (article about CRISPR and AI)
  • HighTech.plus (article about CRISPR therapy)
  • Doctor.rambler.ru (article about genetic scissors)
  • KP.ua (medical breakthroughs 2025)
  • Medgorod-clinic.ru (article about CRISPR and genome editing)

Sources: