Gene Editing Glossary
Every scientific term you'll encounter on this site, explained simply. Hover on highlighted terms in any article for instant definitions.
Biology
fetal hemoglobin
A form of hemoglobin (HbF) naturally produced during fetal development but largely silenced after birth. Reactivating fetal hemoglobin via CRISPR editing of BCL11A is the mechanism behind Casgevy's treatment for sickle cell disease.
Learn morehemoglobin
The protein in red blood cells that carries oxygen throughout the body. Mutations in hemoglobin genes cause sickle cell disease and beta-thalassemia.
Clinical & Regulatory
accelerated approval
An FDA pathway that allows earlier approval of drugs for serious conditions based on a surrogate endpoint (like biomarker changes) rather than clinical outcomes. Requires confirmatory post-market studies.
BLA
Biologics License Application — the application submitted to the FDA for approval to market a biological product (including gene therapies). Equivalent to an NDA for traditional drugs.
Learn moreBreakthrough Therapy
An FDA designation for drugs that treat serious conditions where preliminary evidence shows substantial improvement over existing treatments. Provides intensive FDA guidance and may speed development and review.
DSMB
Data Safety Monitoring Board — an independent committee that reviews ongoing clinical trial data to ensure patient safety. Can recommend stopping a trial early for safety concerns or overwhelming efficacy.
IND
Investigational New Drug application — a request submitted to the FDA before a new drug or therapy can be tested in human clinical trials. Includes preclinical safety data, manufacturing information, and the proposed trial design.
Learn moreinformed consent
The process by which clinical trial participants are fully informed about the study's purpose, procedures, risks, and benefits before agreeing to participate. A legal and ethical requirement in all human research.
PDUFA date
Prescription Drug User Fee Act date — the FDA's deadline to complete its review of a drug or biologic application. Missing a PDUFA date is unusual and typically signals regulatory complications.
Phase 1 trial
The first stage of human clinical testing, primarily assessing safety, dosing, and side effects in a small group of participants (typically 20-80). For gene therapies, Phase 1 often also measures early signs of efficacy.
Learn morePhase 2 trial
The second stage of clinical testing, evaluating efficacy and further assessing safety in a larger group (typically 100-300 participants). Many gene therapy trials combine Phase 1 and 2 into Phase 1/2 studies.
Learn morePhase 3 trial
Large-scale clinical trials (hundreds to thousands of participants) designed to confirm efficacy, monitor side effects, and compare to standard treatments. Successful Phase 3 results typically support regulatory approval.
Learn moreRMAT designation
Regenerative Medicine Advanced Therapy designation — an FDA program that provides expedited review, early interactions with FDA, and potential accelerated approval for regenerative medicine therapies including gene therapies.
surrogate endpoint
A measurable outcome in a clinical trial (such as hemoglobin levels or biomarker changes) used as a stand-in for a clinical outcome like survival. Allows faster drug approval but requires later confirmation of real-world benefit.
Diseases
AATD
Alpha-1 Antitrypsin Deficiency — a genetic condition where the liver produces insufficient or abnormal alpha-1 antitrypsin protein, leading to lung and liver disease. Gene augmentation and editing therapies are in development.
ATTR amyloidosis
Transthyretin amyloidosis — a progressive disease where misfolded TTR protein accumulates in organs, causing nerve and heart damage. Intellia Therapeutics' CRISPR-based in vivo therapy (NTLA-2001) knocks out the TTR gene in liver cells.
beta-thalassemia
A genetic blood disorder caused by mutations in the beta-globin gene, resulting in reduced or absent hemoglobin production. Patients require regular blood transfusions. Casgevy is also approved for transfusion-dependent beta-thalassemia.
Learn moreCGD
Chronic Granulomatous Disease — a rare inherited immune disorder where white blood cells cannot kill certain bacteria and fungi, causing recurrent severe infections. Gene therapy approaches aim to restore the missing enzyme in blood stem cells.
cystic fibrosis
A genetic disorder caused by mutations in the CFTR gene, leading to thick, sticky mucus in the lungs and other organs. Gene editing and gene therapy approaches aim to deliver a corrected CFTR gene to lung cells, a major delivery challenge.
Learn moreDuchenne muscular dystrophy
A severe X-linked genetic disorder caused by mutations in the dystrophin gene, leading to progressive muscle weakness and degeneration. Gene therapy approaches include micro-dystrophin delivery (Elevidys) and exon skipping.
Learn moreHAE
Hereditary Angioedema — a rare genetic disorder causing recurrent episodes of severe swelling. Caused by mutations affecting the KLKB1 pathway. Intellia's in vivo CRISPR therapy targets the KLKB1 gene in the liver to prevent attacks.
Learn morehemophilia
A genetic bleeding disorder caused by deficiency in clotting factor VIII (hemophilia A) or IX (hemophilia B). Gene therapy (Hemgenix for hemophilia B) can deliver a working copy of the clotting factor gene to the liver.
Learn moreLCA10
Leber Congenital Amaurosis type 10 — a rare inherited retinal disease caused by mutations in the CEP290 gene, leading to severe vision loss in childhood. Editas Medicine's EDIT-101 was the first in vivo CRISPR therapy tested in patients.
Learn moresickle cell disease
A genetic blood disorder caused by a single point mutation in the hemoglobin gene, producing abnormal hemoglobin that distorts red blood cells into a sickle shape. Causes severe pain crises, organ damage, and shortened lifespan. The first disease treated with CRISPR (Casgevy).
Learn moreSMA
Spinal Muscular Atrophy — a genetic neuromuscular disease caused by mutations in the SMN1 gene, leading to motor neuron loss and muscle weakness. Treated with the gene therapy Zolgensma, which delivers a functional SMN1 gene via AAV9.
Learn moreGene Editing
ABE
Adenine Base Editor — a base editing tool that converts A-T base pairs to G-C. Uses an evolved adenosine deaminase fused to nickase Cas9. Together with CBEs, ABEs can correct roughly 60% of disease-causing point mutations.
Learn moreADAR
Adenosine Deaminase Acting on RNA — a natural enzyme that converts adenosine (A) to inosine (I) in RNA, which the cell reads as guanosine (G). Harnessed for programmable RNA editing without altering DNA.
Learn morebase editing
A precision gene editing technique that chemically converts one DNA base into another without cutting the double helix. Can correct ~60% of known disease-causing point mutations.
Learn moreCas12a
A CRISPR-associated protein (also called Cpf1) that cuts DNA using a single guide RNA. Unlike Cas9, Cas12a creates staggered cuts and recognizes T-rich PAM sequences, expanding the range of targetable sites.
Cas13
A CRISPR protein that targets and cuts RNA instead of DNA. Used for RNA knockdown, diagnostics (SHERLOCK), and RNA editing applications without permanently altering the genome.
Learn moreCas9
The molecular 'scissors' protein used in CRISPR gene editing. Cas9 cuts both strands of DNA at the location specified by the guide RNA.
CasMINI
An engineered, ultra-compact Cas protein roughly half the size of Cas9. Its small size makes it especially attractive for AAV-based delivery, where cargo capacity is limited to ~4.7 kb.
CasX
A compact CRISPR protein discovered in groundwater bacteria. Smaller than Cas9, making it easier to package in AAV vectors for gene therapy delivery. Licensed by Scribe Therapeutics.
CBE
Cytosine Base Editor — a base editing tool that converts C-G base pairs to T-A without cutting the DNA. Combines a nickase Cas9 with a cytidine deaminase enzyme. Developed by David Liu's lab.
Learn moreCRISPR
Clustered Regularly Interspaced Short Palindromic Repeats — a natural bacterial immune system repurposed as a precise gene editing tool. Uses a guide RNA to direct the Cas9 protein to cut DNA at specific locations.
Learn moreCRISPRa
CRISPR Activation — using dCas9 fused to transcriptional activators to turn on gene expression without cutting DNA. A powerful research tool also being explored therapeutically to upregulate beneficial genes.
CRISPRi
CRISPR Interference — using dCas9 fused to transcriptional repressors to silence gene expression without altering the DNA sequence. Offers a reversible alternative to gene knockout.
dCas9
Dead Cas9 — a catalytically inactive version of Cas9 that binds DNA without cutting it. Used as a programmable platform for gene activation (CRISPRa), repression (CRISPRi), and epigenetic editing.
Learn moredeaminase
An enzyme that removes an amino group from a DNA base, chemically converting it to a different base. The key enzyme in base editing — CBEs use cytidine deaminases, ABEs use adenosine deaminases.
DETECTR
DNA Endonuclease-Targeted CRISPR Trans Reporter — a CRISPR-Cas12a-based diagnostic platform developed by Mammoth Biosciences. Provides rapid, accurate detection of specific DNA sequences for point-of-care diagnostics.
Learn moredouble-strand break
A cut through both strands of the DNA double helix. Standard CRISPR-Cas9 creates DSBs, which the cell repairs — sometimes imperfectly, causing insertions or deletions (indels).
DSB
Double-Strand Break — a cut through both strands of the DNA helix. Traditional CRISPR-Cas9 editing creates DSBs, which the cell repairs via NHEJ or HDR. Newer techniques like base and prime editing avoid DSBs to improve safety.
epigenetic editing
Modifying gene expression without changing the DNA sequence itself, by adding or removing chemical marks like methyl groups or histone modifications. Uses dCas9 fused to epigenetic enzymes to turn genes on or off.
Learn moregene drive
A CRISPR-based genetic engineering technique that biases inheritance so a modified gene spreads through a wild population faster than normal. Studied for controlling malaria-carrying mosquitoes, but raises ecological concerns.
gene knockout
Completely disabling a gene so it no longer produces a functional protein. CRISPR achieves this by creating indels that disrupt the gene's reading frame. Used both in research and therapeutically (e.g., knocking out BCL11A or TTR).
germline editing
Genetic modifications made to eggs, sperm, or embryos that would be inherited by future generations. Currently prohibited in most countries for clinical use due to ethical concerns. Distinct from somatic editing, which affects only the treated individual.
Learn moreguide RNA
A short RNA molecule (~20 nucleotides) that directs the Cas9 protein to the correct location in the genome. Designed to match the target DNA sequence.
HDR
Homology-Directed Repair — a precise but less efficient DNA repair pathway that uses a template to insert a specific sequence at a double-strand break. Requires cells to be dividing, limiting its use in many tissues.
indels
Insertions or deletions of DNA bases that occur when the cell repairs a double-strand break. Can disrupt gene function — sometimes intentionally (gene knockout) or unintentionally (off-target damage).
multiplexed editing
Editing multiple genes or genomic sites simultaneously using multiple guide RNAs in a single experiment. Enables complex genetic modifications and is used in research, agriculture, and some therapeutic approaches.
NHEJ
Non-Homologous End Joining — the cell's default, error-prone repair pathway for double-strand breaks. Often introduces small insertions or deletions (indels), making it useful for gene knockouts but less precise than HDR.
nickase
A modified Cas9 that cuts only one strand of the DNA double helix instead of both. Used in base editing and prime editing to reduce off-target effects compared to full double-strand breaks.
off-target effects
Unintended edits at DNA locations similar to the target site. A major safety concern in gene editing that researchers work to minimize through improved guide RNA design and high-fidelity Cas9 variants.
PAM
Protospacer Adjacent Motif — a short DNA sequence (usually NGG for SpCas9) required next to the target site for Cas9 to bind and cut. Limits which sites can be edited.
pegRNA
Prime Editing Guide RNA — a specialized guide RNA used in prime editing that contains both a target-binding sequence and a template for the desired edit. Directs the prime editor to the correct site and provides the replacement sequence.
Learn moreprime editing
A 'search-and-replace' gene editing technique that can make all 12 possible base changes plus small insertions and deletions without double-strand breaks. Invented by David Liu in 2019.
protospacer
The target DNA sequence complementary to the spacer in the guide RNA. The protospacer must be adjacent to a PAM sequence for Cas9 to recognize and cleave the site.
reverse transcriptase
An enzyme that synthesizes DNA from an RNA template. In prime editing, a reverse transcriptase is fused to nickase Cas9 to write new genetic information directly into the genome using the pegRNA as a template.
Learn moreRNA editing
Altering RNA molecules after they are transcribed from DNA, changing the protein they encode without permanently modifying the genome. Offers a reversible alternative to DNA editing.
Learn moreSHERLOCK
Specific High-sensitivity Enzymatic Reporter un-LOCKing — a CRISPR-Cas13-based diagnostic tool that detects specific RNA sequences with high sensitivity. Used for rapid detection of viruses, cancer mutations, and genetic diseases.
somatic editing
Gene editing applied to non-reproductive body cells. Changes affect only the treated individual and are not passed to offspring. All currently approved gene therapies and CRISPR treatments use somatic editing.
spacer sequence
The ~20-nucleotide portion of a guide RNA that is complementary to the target DNA sequence. Determines where Cas9 or other CRISPR proteins will bind and cut.
Gene Therapy
AAV
Adeno-Associated Virus — a small, non-pathogenic virus commonly used to deliver therapeutic genes into cells. The most widely used vector in gene therapy clinical trials.
capsid
The protein shell of a virus or viral vector. AAV capsid engineering is a major area of research, as different capsids determine which tissues the vector can reach (tropism) and how the immune system responds.
Learn moreCAR-T cell therapy
Chimeric Antigen Receptor T-cell therapy — an immunotherapy that engineers a patient's T cells to recognize and attack cancer cells. Uses lentiviral or CRISPR-based approaches to add a synthetic receptor gene to the T cells.
Learn moreCasgevy
The first CRISPR-based gene therapy approved by the FDA (December 2023). Treats sickle cell disease and beta-thalassemia by editing the BCL11A gene to reactivate fetal hemoglobin.
Learn moreelectroporation
A delivery technique that uses brief electrical pulses to create temporary pores in cell membranes, allowing gene editing components (like RNPs) to enter. Widely used in ex vivo therapies.
Elevidys
An AAV-based gene therapy for Duchenne muscular dystrophy that delivers a shortened version of the dystrophin gene (micro-dystrophin). Received accelerated approval from the FDA to treat ambulatory DMD patients.
Learn moreex vivo
Editing performed outside the body. Cells are removed from the patient, edited in the lab, and then infused back. Used by Casgevy for sickle cell disease treatment.
gene therapy
A medical approach that treats disease by adding, replacing, or modifying genetic material inside a patient's cells. Can use viral vectors (AAV, lentivirus) or non-viral delivery methods.
Learn moreHemgenix
An AAV5-based gene therapy for hemophilia B approved by the FDA in 2022. Delivers a functional copy of the Factor IX gene to the liver, significantly reducing or eliminating the need for clotting factor infusions.
Learn morein vivo
Editing performed directly inside the patient's body, typically by injecting the editing machinery (often in lipid nanoparticles) into the bloodstream. Intellia and Verve use this approach.
lentivirus
A type of retrovirus used to deliver genes into cells by integrating them into the host genome. Commonly used in ex vivo gene therapies like CAR-T cell manufacturing, where permanent gene insertion is desired.
lipid nanoparticle
A tiny fat-based sphere used to deliver gene editing tools (mRNA, guide RNA) into cells. The same technology used in COVID-19 mRNA vaccines. Key delivery vehicle for in vivo gene editing.
Luxturna
An AAV2-based gene therapy for inherited retinal dystrophy caused by RPE65 mutations. Approved by the FDA in 2017, it was one of the first gene therapies approved in the US. Delivered by subretinal injection to restore vision.
Learn moreRNP
Ribonucleoprotein — a pre-assembled complex of Cas protein and guide RNA delivered directly into cells. RNPs are active immediately, degrade quickly (reducing off-target risk), and avoid the need for DNA or mRNA encoding the editor.
serotype
A distinct variation of a virus or vector based on its surface proteins. Different AAV serotypes (AAV1, AAV2, AAV5, AAV8, AAV9, etc.) target different tissues — for example, AAV9 crosses the blood-brain barrier.
Learn moretropism
The tendency of a virus or vector to infect specific cell types or tissues. Tropism is determined by the capsid's interaction with cell surface receptors. Engineering tropism is key to targeted gene therapy delivery.
vector
A vehicle used to deliver genetic material into cells. Viral vectors (AAV, lentivirus) and non-viral vectors (lipid nanoparticles, electroporation) are the two main categories used in gene therapy and gene editing.
Learn moreVLP
Virus-Like Particle — a delivery vehicle that mimics the structure of a virus but carries no viral genome. Can be engineered to package and deliver CRISPR components into cells with reduced immunogenicity.
Zolgensma
An AAV9-based gene therapy for spinal muscular atrophy (SMA) that delivers a functional copy of the SMN1 gene. Approved by the FDA in 2019 for children under two. One of the most expensive therapies ever at ~$2.1 million per dose.
Learn moreGenetics
amino acid
The building blocks of proteins. Twenty standard amino acids are encoded by DNA and assembled in specific sequences to create proteins. A single-base mutation can change one amino acid, sometimes causing disease.
chromatin
The complex of DNA and histone proteins that makes up chromosomes. Chromatin can be 'open' (euchromatin, genes accessible) or 'closed' (heterochromatin, genes silenced), controlling gene expression.
codon
A three-nucleotide sequence in mRNA that specifies a particular amino acid (or a stop signal) during protein synthesis. The 64 possible codons map to 20 amino acids, creating redundancy in the genetic code.
Learn moreDNA
Deoxyribonucleic acid — the molecule that carries genetic instructions in all living organisms. Made of two strands twisted into a double helix, with bases A, T, C, and G.
Learn moreenhancer
A regulatory DNA sequence that can increase transcription of a gene, sometimes from a great distance (thousands of base pairs away). Enhancers are key targets for epigenetic editing approaches.
epigenome
The complete set of chemical modifications on DNA and histone proteins that regulate gene expression without changing the underlying DNA sequence. The epigenome changes with age and is a target for longevity research.
Learn moreexon
A segment of a gene that codes for protein. After transcription, exons are spliced together to form the messenger RNA (mRNA) that is translated into protein. Many gene therapies target specific exons — for example, exon skipping in Duchenne muscular dystrophy.
Learn moregene
A segment of DNA that contains instructions for making a specific protein or RNA molecule. Humans have approximately 20,000 protein-coding genes.
gene expression
The process by which information encoded in a gene is used to produce a functional protein or RNA molecule. Involves transcription (DNA to mRNA) and translation (mRNA to protein). Gene editing can modify expression levels.
Learn moregenome
The complete set of DNA in an organism, including all genes and non-coding regions. The human genome contains about 3 billion base pairs.
histone
Proteins around which DNA is wound to form chromatin. Chemical modifications to histones (acetylation, methylation) control how tightly DNA is packed, regulating which genes are accessible for transcription.
intron
A non-coding segment of a gene that is transcribed into RNA but spliced out before translation. Introns were once considered 'junk DNA' but play important roles in gene regulation and alternative splicing.
methylation
The addition of a methyl group (CH3) to DNA, typically at cytosine bases. DNA methylation generally silences gene expression and changes predictably with age, forming the basis of epigenetic clocks.
Learn moremRNA
Messenger RNA — a temporary copy of a gene that carries instructions from DNA in the nucleus to ribosomes in the cytoplasm for protein synthesis. mRNA technology is used in COVID-19 vaccines and as a delivery format for CRISPR components.
Learn moremutation
A change in the DNA sequence. Can be as small as a single base change (point mutation) or involve large segments of DNA. Some mutations cause disease; others are harmless.
nucleotide
The basic building block of DNA and RNA. Each nucleotide contains a sugar, a phosphate group, and one of four bases: adenine (A), thymine (T), cytosine (C), or guanine (G).
point mutation
A change in a single DNA base pair. The most common type of disease-causing mutation. Base editing is specifically designed to correct point mutations.
promoter
A DNA sequence upstream of a gene that serves as a binding site for RNA polymerase and transcription factors, controlling when and how much a gene is expressed. Gene therapists carefully select promoters to control therapeutic gene expression.
ribosome
The cellular machinery that reads mRNA and assembles proteins by linking amino acids together. Ribosomes consist of ribosomal RNA (rRNA) and proteins, and are found in all living cells.
RNA
Ribonucleic acid — a single-stranded molecule similar to DNA that plays multiple roles including carrying genetic instructions (mRNA), forming ribosomes (rRNA), and transferring amino acids (tRNA). Also used as a guide molecule in CRISPR systems.
Learn moretranscription
The process of copying DNA into messenger RNA (mRNA) by the enzyme RNA polymerase. The first step of gene expression, occurring in the cell nucleus.
Learn moretranslation
The process of assembling a protein from amino acids based on the sequence encoded in mRNA. Carried out by ribosomes in the cytoplasm. The second major step of gene expression.
Learn moreLongevity Science
autophagy
The cell's recycling system — a process that breaks down and removes damaged proteins and organelles. Declines with age. Stimulated by fasting, exercise, and mTOR inhibition, and believed to be important for healthy aging.
biological age
A measure of how old your body actually is at a molecular level, as opposed to chronological age (years since birth). Estimated using biomarkers like DNA methylation patterns, and can be older or younger than chronological age.
Learn moreepigenetic clock
A molecular test that estimates biological age by measuring DNA methylation patterns at specific sites across the genome. Developed by Steve Horvath and others. Used to measure the effect of anti-aging interventions.
Learn moreepigenetics
The study of changes in gene expression that don't involve changes to the DNA sequence itself. Epigenetic modifications (like DNA methylation) can be inherited and are linked to aging.
Learn morehealthspan
The period of life spent in good health, free from chronic disease and disability. The primary goal of longevity science — extending healthspan matters more than simply extending lifespan.
lifespan
The total length of time an organism lives. Maximum human lifespan is approximately 120 years. Longevity research aims to extend both average and maximum lifespan while maintaining quality of life.
mTOR
Mechanistic Target of Rapamycin — a protein kinase that regulates cell growth, metabolism, and autophagy. Inhibiting mTOR (e.g., with rapamycin) extends lifespan in many organisms and is a leading target in longevity research.
NAD+
Nicotinamide Adenine Dinucleotide — a coenzyme essential for cellular energy production, DNA repair, and sirtuin activation. NAD+ levels decline with age, and boosting them with precursors like NMN or NR is a popular longevity strategy.
Learn morepartial reprogramming
Briefly expressing Yamanaka factors to reset a cell's epigenetic age without fully reverting it to a stem cell state. A promising anti-aging strategy being pursued by companies like Altos Labs and NewLimit.
Learn moresenescent cells
Cells that have permanently stopped dividing but resist dying, accumulating with age. They secrete inflammatory molecules (the SASP) that damage surrounding tissue. Clearing senescent cells is a major anti-aging strategy.
Learn moresenolytics
Drugs that selectively destroy senescent (aged, non-dividing) cells. These 'zombie cells' accumulate with age and contribute to inflammation and tissue damage.
sirtuins
A family of seven proteins (SIRT1-7) involved in regulating cellular health, DNA repair, metabolism, and inflammation. Require NAD+ to function and are central to several theories of aging.
telomerase
An enzyme that extends telomeres, the protective caps on chromosome ends. Most adult cells do not express telomerase, allowing telomeres to shorten with each division. Reactivating telomerase is explored as an anti-aging approach, though it carries cancer risk.
Learn moretelomere
Protective caps at the ends of chromosomes that shorten with each cell division. Telomere length is a biomarker of cellular aging — shorter telomeres are associated with age-related diseases.
Yamanaka factors
Four transcription factors (Oct4, Sox2, Klf4, c-Myc) that can reprogram adult cells back to a pluripotent stem cell state. Partial reprogramming with these factors is being explored to reverse aging without causing tumor formation.
Learn moreStem Cells & Regenerative Medicine
bioprinting
Using 3D printing technology to deposit living cells, biomaterials, and growth factors layer by layer to fabricate tissue-like structures. An emerging approach in regenerative medicine for creating transplantable tissues and organs.
differentiation
The process by which a stem cell becomes a specialized cell type (neuron, blood cell, muscle cell, etc.). Directed differentiation of gene-edited iPSCs is a key strategy for creating cell therapies.
HSPCs
Hematopoietic Stem and Progenitor Cells — blood-forming stem cells found in bone marrow that give rise to all blood cell types. The target cells for gene therapies treating blood disorders like sickle cell disease, beta-thalassemia, and severe combined immunodeficiency.
iPSCs
Induced Pluripotent Stem Cells — adult cells reprogrammed back to an embryonic-like state using Yamanaka factors (Oct4, Sox2, Klf4, c-Myc). Discovered by Shinya Yamanaka in 2006 (Nobel Prize 2012). Can differentiate into any cell type, enabling patient-specific disease models and cell therapies without embryonic tissue.
Learn moremesenchymal stem cells
Multipotent stem cells that can differentiate into bone, cartilage, fat, and muscle cells. Found in bone marrow, fat tissue, and umbilical cord blood. Widely studied for regenerative medicine, though clinical evidence for many uses remains limited.
organoids
Miniature 3D organ models grown from stem cells in the lab. Recapitulate the architecture and function of real organs (brain, gut, liver, kidney). Used to study disease, test drugs, and model gene editing effects before clinical use.
Learn morepluripotency
The ability of a stem cell to develop into any cell type in the body. Embryonic stem cells and iPSCs are pluripotent. Partial reprogramming aims to reset the epigenetic clock without full pluripotency to avoid tumor risk.
stem cells
Unspecialized cells that can self-renew and differentiate into specialized cell types. The foundation of regenerative medicine and a key vehicle for ex vivo gene therapies like Casgevy, which edits a patient's own stem cells.
xenotransplantation
Transplanting organs, tissues, or cells from one species into another — typically CRISPR-edited pig organs into humans. Active research area with pig kidneys, hearts, livers, and lungs transplanted into human patients since 2022.
Synthetic Biology
biofoundry
An automated facility that uses robotics and AI to design, build, and test biological systems at high throughput. Companies like Ginkgo Bioworks operate biofoundries for engineering organisms for industrial applications.
biosecurity
Measures to protect against the misuse of biological research and technology, including engineered pathogens. Increasingly important as gene editing becomes more accessible.
gene circuit
An engineered network of genes and regulatory elements designed to perform a specific logical function inside a cell, such as sensing a disease biomarker and producing a therapeutic protein in response.
synthetic biology
The engineering discipline of designing and building new biological parts, devices, and systems — or redesigning existing natural systems for useful purposes.
Learn more