Anna's Deep Dives

_{Just facts, you think for yourself}

Curing Genetic Diseases: Sickle Cell, Cystic Fibrosis, and Beyond

Gene editing is transforming treatment for genetic diseases. CRISPR-Cas9 allows precise DNA modifications. Researchers have altered 80% of targeted alleles in diseases like sickle cell disease and β-thalassemia without unintended effects.

Sickle cell disease (SCD) affects about 100,000 people in the U.S. and millions worldwide, causing severe pain and reducing life expectancy. In 2023, the FDA approved Casgevy, the first CRISPR-based therapy for SCD. Clinical trials showed 96.6% of treated patients experienced fewer pain crises, with many becoming pain-free.

Casgevy modifies blood stem cells to produce healthy hemoglobin. The UK’s National Health Service approved it for patients over 12 years old in 2025. The treatment costs around £1.65 million but provides an option for those without a stem cell donor. Scientists at UCSF Benioff Children's Hospital are testing CRISPR to directly correct sickle cell mutations in blood stem cells.

Cystic fibrosis (CF) affects about 40,000 people in the U.S., damaging the lungs and digestive system. Mutations in the CFTR gene disrupt salt and water balance in cells, leading to thick mucus buildup.

Gene therapy aims to correct CFTR mutations. Restoring just 5-10% of normal CFTR function can improve lung health. Scientists are testing viral vectors and lipid nanoparticles to deliver corrective genes. CRISPR-based therapy has successfully corrected CF mutations in lung stem cells in animal models.

Other genetic diseases, including phenylketonuria and familial hypercholesterolemia, are being studied for gene editing treatments. Prime editing and base editing offer precise methods for correcting mutations without cutting DNA. These approaches have already corrected mutations linked to metabolic disorders.

Hearing loss is another target for gene therapy. In 2024, doctors restored hearing in an 18-month-old girl using a virus carrying the OTOF gene. Within 24 weeks, her hearing reached near-normal levels, offering hope to millions with sensorineural hearing loss.

Despite progress, challenges remain. Gene therapies cost between $450,000 and $3 million, limiting accessibility. Ethical concerns over unintended mutations and germline editing persist.

Researchers are improving delivery methods and ensuring safety. The U.S. genome editing market, valued at $3.55 billion in 2023, is projected to reach $16.49 billion by 2033. Continued research may provide lasting cures for many genetic diseases.

The Promise of Cancer Treatments

Gene editing is reshaping cancer treatment. CRISPR-Cas9 targets genes involved in cancer growth. Over 25 clinical trials are testing CRISPR-based approaches against various cancers.

CAR-T cell therapy reprograms a patient’s T-cells to attack cancer, achieving a 40% remission rate in certain blood cancers. Researchers aim to expand CAR-T therapy to solid tumors, which are harder to treat.

mRNA vaccines show promise, with over 100 clinical trials testing them for breast cancer, melanoma, and other tumors. Scientists are developing self-amplifying mRNA vaccines to enhance immune response and durability.

Challenges include reducing off-target effects and improving delivery methods. Scientists are refining nanoparticle and viral vector systems to ensure edited genes reach the right cells.

New gene editing tools, such as base editing and prime editing, improve precision and reduce unintended mutations. These advancements may lead to personalized cancer treatments in the near future.

Organ Transplants & Xenotransplantation (Editing Pig Organs for Humans)

More than 100,000 people in the U.S. need organ transplants. Every day, about 17 patients die waiting for a donor. Scientists are exploring xenotransplantation—using animal organs for human transplants—as a potential solution.

Pigs are the best candidates due to their organ size and function. However, pig organs trigger immediate rejection in humans. CRISPR-Cas9 removes rejection-causing pig genes and adds human genes for compatibility. Some pig kidneys now have up to 10 genetic edits.

In March 2024, doctors transplanted a gene-edited pig kidney into a human patient, who survived for two months before passing away from unrelated causes. The procedure showed pig organs can function in humans.

In February 2025, the FDA approved a clinical trial for xenotransplantation. Researchers are testing pig kidney transplants in patients with chronic kidney disease, affecting over 90,000 people on waiting lists.

Towana Looney, a 53-year-old dialysis patient, became the first to receive a gene-edited pig kidney in a clinical trial. The organ, with 10 genetic modifications, functioned well months after surgery.

Challenges remain. The biggest risk is immune rejection. Scientists are refining gene edits for better compatibility. They also aim to prevent zoonotic diseases, infections that could transfer from pigs to humans.

Ethical concerns include the use of animals for organ harvesting and potential health risks. Researchers and regulators seek to balance medical progress with ethical responsibility.

If successful, gene-edited pig organs could help thousands of patients who would otherwise die waiting for a transplant. Ongoing research could make xenotransplantation a viable solution.

The Emerging Field of Epigenetic Editing

Epigenetic editing controls genes without changing their DNA sequence. Instead of cutting or replacing genes, it adds or removes chemical tags to turn genes on or off. This allows scientists to adjust gene activity without permanent DNA alterations.

DNA methylation is a key mechanism. Methyl groups silence certain genes. Removing them can reactivate genes. Scientists use CRISPR-based tools like dCas9-SDD to modify methyl groups with precision.

Epigenetic editing has shown potential for treating Angelman syndrome, a neurological disorder caused by an inactive gene. Targeted methylation successfully reactivated the gene in lab models, with only 2 out of 15 possible off-target sites affected.

Another breakthrough involves prion diseases, which cause severe brain damage. Researchers developed CHARMs, a method that silences harmful prion proteins. Lab tests showed an 80% reduction in prion proteins, suggesting a promising treatment.

Cancer research is exploring epigenetic editing. Many cancers involve hypermethylation of tumor-suppressor genes, switching them off. Reversing these changes may reactivate protective genes and slow cancer growth. In 2023, over 20 million new cancer cases were linked to epigenetic abnormalities.

The global epigenetics market is growing. By 2033, it is expected to reach $61 billion, with North America leading research and investment. New tools like single-cell epigenomics are advancing understanding of epigenetic influences on disease.

Challenges include improving delivery methods and understanding long-term effects. Ethical concerns exist, particularly regarding heritable changes in epigenetic modifications.

Epigenetic editing offers a way to treat diseases without permanently altering DNA. As research advances, it may provide new treatments for genetic disorders, cancer, and neurological diseases. The ability to switch genes on and off with precision could transform medicine.

We don’t take shortcuts, chase headlines, or push narratives. We just bring you the news, straight and fair. If you value that, click here to become a paid subscriber—your support makes all the difference.

Table of Contents

(Click on any section to start reading it)

1. Introduction

Why Gene Editing Matters

2. The Science of Gene Editing

DNA 101: The Blueprint of Life

How Mutations Shape Evolution & Disease

Early Gene Editing Techniques: The Precursors to CRISPR

CRISPR & Beyond: How Gene Editing Works Today

3. The History of Gene Editing

The Discovery of DNA & the Genetic Code

The Rise of Genetic Engineering: From GMOs to Gene Therapy

CRISPR’s Breakthrough: A Nobel Prize-Winning Revolution

4. The Applications of Gene Editing

Medicine & Human Health

Curing Genetic Diseases: Sickle Cell, Cystic Fibrosis, and Beyond

The Promise of Cancer Treatments

Organ Transplants & Xenotransplantation (Editing Pig Organs for Humans)

The Emerging Field of Epigenetic Editing

Agriculture & Food Production

Genetically Modified Crops vs. CRISPR-Edited Crops

Engineering Disease-Resistant Livestock

The Fight Against Food Insecurity & Climate Change

Biotechnology & Synthetic Biology

Gene Editing for Drug Development

Biomanufacturing: Editing Bacteria to Produce Medicine

Engineering New Life Forms

5. The Business of Gene Editing

The Race for Patents: Who Owns CRISPR?

The Leading Gene Editing Companies & Their Technologies

The Economics of Gene Therapies: Can We Make It Affordable?

Investing in Gene Editing: Risks & Opportunities

6. The Ethical Dilemmas of Gene Editing 

Designer Babies & Human Enhancement

The Risk of Eugenics & Genetic Discrimination

Should We Edit the Human Germline?

Regulating a Global Technology: Who Decides What’s Ethical?

7. The Risks & Challenges of Gene Editing

Off-Target Effects & Unintended Consequences

Biosecurity Concerns: Could CRISPR Be Weaponized?

The Challenge of Public Acceptance & Misinformation

8. The Future of Gene Editing

CRISPR 2.0 & Next-Gen Technologies

The Path to Curing All Genetic Diseases

The Ultimate Ethical Dilemma: Should We Edit Ourselves to Evolve?

Baked with love,

Anna Eisenberg ❤️