La mappa della rivoluzione dell'editing genico
Il CRISPR Atlas
Esplora le tecnologie, i trattamenti, le malattie, gli scienziati, le aziende, i trial clinici e le scoperte che stanno plasmando il futuro della medicina genetica.
Esplora il gene editing
Sette modi in cui gli scienziati modificano il materiale genetico. Ogni pagina si apre con una spiegazione in linguaggio semplice, poi approfondisce quanto vuoi.
CRISPR-Cas9A protein that can be programmed with a short RNA guide to find one specific sequence in a genome and cut it.
Base EditingChemically converts one DNA letter into another at a chosen position, without cutting both strands of the double helix.
Prime EditingWrites a new stretch of DNA sequence directly into a chosen site, using a guide that carries the replacement text with it.
CRISPR-Cas12A family of CRISPR nucleases that cut DNA leaving staggered ends, use a different sequence requirement from Cas9, and can be smaller.
CRISPR-Cas13Targets RNA rather than DNA, so it can silence a gene's message without touching the genome itself.
Cas14 and miniature Cas systemsUnusually small CRISPR nucleases — roughly a third the size of Cas9 — discovered in archaea and bacteriophages.
RNA EditingChanges letters in the RNA message rather than in the DNA, so the effect is real but temporary and the genome is untouched.
Confrontale affiancatePrecisione, taglio, dimensione del carico, somministrazione, maturità, rischi
Malattie prese di mira
Ogni pagina dedicata a una malattia indica chiaramente se il gene editing è un trattamento approvato, in fase di sperimentazione o ancora solo un'idea di laboratorio.
Sickle Cell DiseaseBlood disorders
Beta ThalassemiaBlood disorders
ATTR AmyloidosisMetabolic disease
Hereditary AngioedemaRare genetic disease
High Cholesterol and Familial HypercholesterolemiaCardiovascular disease
CancerCancer
Leukemia and LymphomaCancer
HIVInfectious disease
Duchenne Muscular DystrophyMuscular disease
Cystic FibrosisRare genetic disease
Trattamenti
Farmaci con nome proprio, con lo stato regolatorio indicato in ogni scheda.
Farmaci di editing genico approvati nell'Atlas: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)
Sperimentazioni cliniche
Dove vengono prodotte davvero le prove scientifiche.
Aziende da conoscere
Chi sta costruendo cosa e a che punto è ciascun programma.
CRSP · Zug, Switzerland
CRISPR Therapeutics
Co-founded by Emmanuelle Charpentier, and the company behind the first approved CRISPR…
NTLA · Cambridge, Massachusetts
Intellia Therapeutics
Co-founded by Jennifer Doudna, and the company that first delivered CRISPR into a human…
BEAM · Cambridge, Massachusetts
Beam Therapeutics
Founded by David Liu and colleagues to commercialise base editing — precision changes to…
EDIT · Cambridge, Massachusetts
Editas Medicine
One of the first CRISPR companies, which discontinued its cell-therapy and eye programmes…
PRME · Cambridge, Massachusetts
Prime Medicine
Founded to commercialise prime editing, and the company behind the first published…
Privato · Boston, Massachusetts
Verve Therapeutics
Built to turn heart disease into a one-time treatment, and acquired by Eli Lilly in 2025…
CRBU · Berkeley, California
Caribou Biosciences
Co-founded by Jennifer Doudna, using hybrid RNA-DNA guides intended to make editing…
SANA · Seattle, Washington
Sana Biotechnology
Engineering transplanted cells to be invisible to the immune system, so they can be given…
Scienziati che hanno cambiato il campo
Il contributo effettivo di ogni persona — l'attribuzione dei meriti in questo campo è controversa, e lo diciamo chiaramente.
Jennifer DoudnaStructural biochemist who, with Emmanuelle Charpentier, showed that Cas9 could be programmed with a single guide RNA to cut chosen DNA — and shared the 2020 Nobel Prize in Chemistry for it.
Emmanuelle CharpentierMicrobiologist who discovered tracrRNA, the missing component of the CRISPR-Cas9 system, and shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna.
Feng ZhangBioengineer who led one of the first demonstrations of CRISPR genome editing in human cells and discovered several new CRISPR systems including Cas12a and Cas13.
David LiuChemical biologist whose laboratory invented base editing and prime editing — the two techniques that changed gene editing from cutting to writing.
George ChurchGeneticist whose laboratory independently demonstrated CRISPR editing in human cells in 2013, and a founder of an unusually large number of genomics companies.
Virginijus ŠikšnysLithuanian biochemist who independently showed that Cas9 could be programmed to cut DNA, publishing in 2012 — and whose contribution is often omitted from popular accounts.
Ultime notizie sul gene editing
Articoli reali da editori identificati. Ogni titolo rimanda all'originale — non riscriviamo né ripubblichiamo mai i contenuti.
CRISPR spiegato
Inizia dall'inizio. Non è necessaria una preparazione in biologia.
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What is DNA?
DNA is a long chemical chain that stores the instructions for building and running a living thing, written in…
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What is a gene?
A gene is a stretch of DNA that carries the instructions for making one particular thing the cell needs —…
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What is a chromosome?
A chromosome is one very long DNA molecule packaged with proteins; humans normally have 46, in 23 pairs.
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What is a genome?
A genome is the complete set of genetic instructions in an organism — every chromosome, every gene, and…
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What is RNA?
RNA is DNA's working copy — a temporary, single-stranded version the cell makes when it needs to use a gene.
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What is a mutation?
A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are beneficial.
Il gene editing in numeri
Conteggi in tempo reale dal database dell'Atlante — questi sono il numero di schede che abbiamo, non una stima delle dimensioni del settore.
I conteggi si aggiornano a ogni caricamento della pagina. "Approvato" significa che un'autorità regolatoria nazionale ha autorizzato almeno un prodotto; non significa che la terapia sia disponibile o adatta a una determinata persona.
