O guia definitivo sobre edição genética.
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É novo em genética? Explicamos cada termo à medida que navega, em linguagem simples. As mesmas páginas, com a ajuda integrada.

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Você já conhece a biologia. Apenas o conteúdo — limpo e compacto, sem explicações extras. Esta é a visualização padrão.

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O mapa da revolução da edição genética

O CRISPR Atlas

Explore as tecnologias, tratamentos, doenças, cientistas, empresas, ensaios clínicos e descobertas que estão a moldar o futuro da medicina genética.

17 tecnologias 19 doenças 15 treatments 14 clinical trials 17 companies 19 genes Referência educativa · apenas para pesquisa · nunca aconselhamento médico
You are in Guided View. Terms are explained as you go, and every page opens with a plain-language summary before the technical detail. Switch back to Expert View from the menu at any time.

Start where you actually are

This site is written for very different readers at once. Each door below leads into the same database from a different angle — nothing is dumbed down, and nothing assumes a biology degree.

New to all of thisStart with what DNA is and build up. No background needed. A patient or familyWhat is approved, what is only in trials, and how to tell the difference. Following the industryWho is developing what, at which stage. Information, never advice. Here for food and farmingGene-edited crops and livestock, and why the rules differ by country.

Explorar a edição genética

Sete formas como os cientistas modificam o material genético. Cada página começa com uma explicação em linguagem simples e vai tão fundo quanto quiser.

Todas as tecnologias →
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. Compare-as lado a ladoPrecisão, corte, tamanho da carga, entrega, maturidade, riscos

Doenças sendo tratadas

Cada página de doença indica claramente se a edição genética é um tratamento aprovado, está em ensaios clínicos ou ainda é apenas uma ideia de laboratório.

Todas as doenças →
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

Tratamentos

Medicamentos identificados pelo nome, com o estado regulatório indicado em cada registo.

Casgevy (exagamglogene autotemcel)The first CRISPR-based medicine approved anywhere in the world — a one-time therapy made from a patient's own edited blood stem cells. Approved Lyfgenia (lovotibeglogene autotemcel)Approved on the same day as Casgevy for sickle cell disease — but a gene-addition therapy, not gene editing, which makes the pair a useful comparison. Approved Zynteglo (betibeglogene autotemcel)An approved gene-addition therapy for transfusion-dependent beta thalassemia — the non-editing alternative to Casgevy in the same disease. Approved Nexiguran ziclumeran (nex-z)The first CRISPR therapy ever administered into a human bloodstream — a single infusion intended to switch off the TTR gene in the liver for good. Phase III Lonvoguran ziclumeran (lonvo-z)The first in vivo gene-editing therapy to report a positive Phase 3 result — a single infusion intended to stop hereditary angioedema attacks permanently. Phase III Risto-cel (BEAM-101)A base-editing therapy for sickle cell disease that mimics a naturally protective genetic variant, with a licence application signalled for as early as the end of 2026. Phase III

Medicamentos de edição génica aprovados no Atlas: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)

Todos os tratamentos →

Ensaios clínicos

Onde as evidências são realmente produzidas.

4A recrutar
2Ativo, sem recrutamento
7Concluído
CLIMB SCD-121 — the trial that led to the first CRISPR medicine approvalSickle cell disease · NCT03745287 Completed CLIMB THAL-111 — the beta thalassemia trial behind the Casgevy approvalBeta thalassemia · NCT03655678 Completed MAGNITUDE — Phase 3 in vivo CRISPR for ATTR cardiomyopathyATTR amyloidosis · NCT06128629 Active, not recruiting MAGNITUDE-2 — Phase 3 in vivo CRISPR for ATTR polyneuropathyATTR amyloidosis · NCT06672237 Paused HAELO — the first positive Phase 3 result for in vivo gene editingHereditary angioedema · NCT06634420 Completed BEACON — base editing for sickle cell diseaseSickle cell disease · NCT05456880 Active, not recruiting

Pesquisar na base de dados de ensaios →

Empresas para conhecer

Quem está desenvolvendo o quê e em que estágio está cada programa.

Todas as empresas →

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…

Privado · 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…

Cientistas que transformaram a área

O que cada pessoa contribuiu de fato — a atribuição de crédito nessa área é contestada, e indicamos onde.

Todos os cientistas →
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.

Últimas notícias sobre edição genética

Artigos reais de editores identificados. Cada título leva ao original — nunca reescrevemos nem republicamos.

Todas as notícias →
First-in-human pilot trial improves stem cell collection and delivery for sickle cell gene therapy Patients with sickle cell disease are often hesitant to undergo transformative gene therapy—the current lengthy process requires multiple hospital visits… Medical Xpress — Genetics Treatments 1h ago↗ Pioneering Study of Alpha-Gal Syndrome Launched at Martha’s Vineyard Hospital Martha’s Vineyard Hospital launches a two-year study tracking up to 300 residents to understand who develops alpha-gal syndrome after lone star tick… GEN — Genetic Engineering News Research 8h ago↗ Bristol Myers ends deal with Cellares, cell therapy manufacturer to reorg After a “comprehensive evaluation,” Bristol Myers Squibb has pulled out of its partnership with Cellares and the cell therapy manufacturer will… Endpoints News Treatments 9h ago↗ Male Sex Hormones Suppress Lung Allergies by Acting on Neurons in Mice A study in mice found that male sex hormones can suppress allergic responses by promoting sympathetic nerve connections and norepinephrine release in the… GEN — Genetic Engineering News Research 1d ago↗ Regenxbio hit with another clinical hold for Hunter syndrome gene therapy Regenxbio said it no longer expects to file for FDA approval of its Hunter syndrome gene therapy in the near future after the program was slapped with its… Endpoints News FDA 1d ago↗ News on AstraZeneca, Cytokinetics, Novartis — what to look out for at ESC 2026 AstraZeneca, Ionis, Cytokinetics, Novartis and CRISPR Therapeutics, among others, will get a sense of the market potential of their heart drugs when data… Endpoints News Companies 1d ago↗ STAT+: Pharmalittle: We’re reading about Medicaid obesity-drug coverage, a Regenxbio gene therapy, and more President Trump’s pledge to make pricey weight loss drugs widely available for low-income Americans through Medicaid is not panning out STAT Agriculture 1d ago↗ STAT+: FDA pauses Regenxbio gene therapy trial again due to safety concerns The FDA put a hold on a Regenxbio gene therapy trial again after new safety concerns emerged. STAT FDA 1d ago↗

O CRISPR explicado

Comece do início. Não são necessários conhecimentos de biologia.

Comece a aprender →

Start here

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.

Start here

What is a mutation?

A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are beneficial.

A edição genética em números

Contagens em tempo real da base de dados do Atlas — correspondem ao número de registos que temos, não a uma estimativa da dimensão do campo.

0Clinical trials trackedRegistos do Atlas
0Terapias de edição génica aprovadasAutorizado pela FDA e/ou EMA
0Diseases coveredRegistos do Atlas
0EmpresasRegistos do Atlas
0CientistasRegistos do Atlas
0GenesRegistos do Atlas
0TecnologiasRegistos do Atlas
0Connections between recordsArestas do grafo de conhecimento

Os contadores são atualizados a cada carregamento de página. "Aprovado" significa que um regulador nacional de medicamentos autorizou pelo menos um produto; isso não significa que a terapia esteja disponível ou seja adequada para qualquer pessoa em particular.

Perguntar ao Atlas Coloque uma pergunta em linguagem simples. As respostas são construídas apenas a partir dos registos com fontes do próprio site, com ligações de volta às páginas utilizadas. A história da edição genética Da estrutura do DNA ao primeiro medicamento CRISPR aprovado — os acontecimentos que nos trouxeram até aqui.
AgriculturaCulturas, animais e as regras que variam por país ÉticaOs argumentos apresentados por ambos os lados InvestimentoComo este setor é financiado — informação, não aconselhamento Mapa mundialOnde a edição genética acontece
Antes de continuar a ler O CRISPR Atlas é uma referência educativa. Não presta aconselhamento médico, e a informação sobre uma terapia experimental nunca é prova de que essa terapia é segura ou eficaz. O estado dos ensaios clínicos e o estado regulatório mudam com frequência. Se estiver em causa uma decisão sobre a sua saúde ou a de um familiar, consulte um profissional de saúde qualificado. Leia os avisos completos →