La guía definitiva sobre edición genética.
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Vista guiada
¿Eres nuevo en genética? Explicamos cada término mientras navegas, en un lenguaje sencillo. Las mismas páginas, con la ayuda incorporada.

Opinión experta
Ya conoces la biología. Solo el contenido, limpio y compacto, sin explicaciones adicionales. Esta es la vista predeterminada.

Idioma de la interfaz
Modo claro
A lone figure before a wall of streaming light

El mapa de la revolución de la edición genética

El CRISPR Decoded

Explora las tecnologías, tratamientos, enfermedades, científicos, empresas, ensayos clínicos y descubrimientos que están dando forma al futuro de la medicina genética.

15 treatments tracked 14 clinical trials Referencia educativa · solo para investigación · nunca consejo 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.

Empieza donde realmente estás

Este sitio está escrito para lectores muy distintos a la vez. Cada puerta de abajo accede a la misma base de datos desde un ángulo diferente. Nada está simplificado en exceso y nada da por sentado que tienes una carrera en biología.

A child's hands holding a small glowing DNA ladder New to all of thisComienza con qué es el DNA y ve avanzando. No se necesitan conocimientos previos. A doctor and a patient talking across a desk A patient or familyQué está aprobado, qué solo está en ensayos y cómo distinguirlo. Nested glowing rectangles receding into darkness Siguiendo a la industriaQuién desarrolla qué y en qué etapa. Información, nunca consejo médico. Rows of seedlings under purple grow lights Aquí por la alimentación y la agriculturaCultivos y animales editados genéticamente, y por qué las normas varían según el país.

Explorar la edición genética

Siete formas en que los científicos modifican el material genético. Cada página comienza con una explicación en lenguaje sencillo y luego profundiza tanto como quieras.

Todas las tecnologías →
the crystal structure of Streptococcus pyogenes Cas9 in complex with sgRNA and its target DNA at 2.5 A ˚ resolution CRISPR-Cas9A protein that can be programmed with a short RNA guide to find one specific sequence in a genome and cut it. Hiroshi Nishimasu, F. Ann Ran,... · CC BY-SA 3.0 A DNA helix with one letter being chemically changed Base EditingChemically converts one DNA letter into another at a chosen position, without cutting both strands of the double helix. A prime editor writing new letters into a DNA strand Prime EditingWrites a new stretch of DNA sequence directly into a chosen site, using a guide that carries the replacement text with it. A Cas12 enzyme on DNA, scattering fragments CRISPR-Cas12A family of CRISPR nucleases that cut DNA leaving staggered ends, use a different sequence requirement from Cas9, and can be smaller. A Cas13 enzyme cutting a strand of RNA CRISPR-Cas13Targets RNA rather than DNA, so it can silence a gene's message without touching the genome itself. A tiny Cas14 enzyme bound to a DNA helix Cas14 and miniature Cas systemsUnusually small CRISPR nucleases, roughly a third the size of Cas9, discovered in archaea and bacteriophages. An enzyme changing a letter on a strand of RNA RNA EditingChanges letters in the RNA message rather than in the DNA, so the effect is real but temporary and the genome is untouched. Two molecular structures side by side, linked by threads of light Compáralas lado a ladoPrecisión, corte, tamaño de carga, administración, madurez, riesgos

Enfermedades en el punto de mira

Cada página de enfermedad indica claramente si la edición génica es un tratamiento aprobado, está en ensayos clínicos o sigue siendo solo una idea de laboratorio.

Todas las enfermedades →
Blood smear from a patient with sickle cell anemia displaying sickle cells, polychromasia, and target cells Sickle Cell DiseaseBlood disorders SpicyMilkBoy · CC BY-SA 4.0 A giemsa stained blood smear from a person with beta thalassemia Beta ThalassemiaBlood disorders Dr Graham Beards · CC BY-SA 4.0 Fibrous amyloid deposits wrapped around heart muscle ATTR AmyloidosisMetabolic disease Swollen right hand in a female patient during a hereditary angioedema attack. Hereditary angioedema Hereditary AngioedemaRare genetic disease LucyHAE · CC BY-SA 3.0 A liver and an artery cross-section with lipoprotein particles High Cholesterol and Familial HypercholesterolemiaCardiovascular disease Micrograph of a nodular basal cell cancer with a cleft. H&E stain. Such clefts are caused by shrinkage of mucin during tissue fixation and staining. Robert S Bader. Which histologi CancerCancer Mikael Häggström, M.D. Author info... · CC0 Blood smear showing acute lymphatic leukaemia (Jenner's stain). Wellcome Images Keywords: Clinical Medicine; Goodall; Haematology; Guilland; Leukaemia Leukemia and LymphomaCancer https://wellcomeimages.org/indexplu... · CC BY 4.0 This scanning electron micrograph revealed the presence of the human immunodeficiency virus (HIV-1), (spherical in appearance), which had been co-cultivated with human lymphocytes. HIVInfectious disease Photo Credit: C. Goldsmith Content... · Public domain Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Cross section of muscle shows extensive replacement of muscle Duchenne Muscular DystrophyMuscular disease Dr. Edwin P. Ewing, Jr. · Public domain A cross-section of an airway clogged with thick mucus Cystic FibrosisRare genetic disease

Tratamientos

Medicamentos con nombre propio, con el estado regulatorio indicado en cada registro.

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 aprobados en CRISPR Decoded: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)

Todos los tratamientos →

Ensayos clínicos

Dónde se genera realmente la evidencia.

4En reclutamiento
2Activo, sin reclutamiento
7Completado
CLIMB SCD-121: the trial that led to the first CRISPR medicine approvalSickle cell disease · NCT03745287 Phase II/III CLIMB THAL-111: the beta thalassemia trial behind the Casgevy approvalBeta thalassemia · NCT03655678 Phase II/III MAGNITUDE: Phase 3 in vivo CRISPR for ATTR cardiomyopathyATTR amyloidosis · NCT06128629 Phase III MAGNITUDE-2: Phase 3 in vivo CRISPR for ATTR polyneuropathyATTR amyloidosis · NCT06672237 Phase III HAELO: the first positive Phase 3 result for in vivo gene editingHereditary angioedema · NCT06634420 Phase III BEACON: base editing for sickle cell diseaseSickle cell disease · NCT05456880 Phase I/II

Buscar en la base de datos de ensayos →

Empresas que debes conocer

Quién está desarrollando qué y en qué punto se encuentra cada programa.

Todas las empresas →
CRISPR TherapeuticsCRSP · Zug, Switzerland Intellia TherapeuticsNTLA · Cambridge, Massachusetts Beam TherapeuticsBEAM · Cambridge, Massachusetts Editas MedicineEDIT · Cambridge, Massachusetts Prime MedicinePRME · Cambridge, Massachusetts Verve TherapeuticsPrivado · Boston, Massachusetts Caribou BiosciencesCRBU · Berkeley, California Sana BiotechnologySANA · Seattle, Washington

Científicos que cambiaron el campo

Lo que cada persona aportó realmente. La atribución de méritos en este campo es debatida, y señalamos dónde.

Todos los científicos →
Jennifer Doudna in 2021 at the Innovative Genomics Institute by Christopher Michel 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. Christopher Michel · CC BY-SA 4.0 Director, Max Planck Institute for Infection Biology, Department of Regulation in Infection Biology. Visiting professor The Laboratory for Molecular Infection Medicine Sweden MIMS; Emmanuelle CharpentierMicrobiologist who discovered tracrRNA, the missing component of the CRISPR-Cas9 system, and shared the 2020 Nobel Prize in Chemistry with Jennifer Doudna. Bianca Fioretti, Hallbauer & Fioretti · CC BY-SA 4.0 Prof. Feng Zhang at the Forbes 30 Under 30 Summit, Boston MA. 2017 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. PuppyEggs · CC BY-SA 4.0 David R. Liu, chemist, 2012 photo David LiuChemical biologist whose laboratory invented base editing and prime editing, the two techniques that changed gene editing from cutting to writing. ServiceAT · CC BY-SA 4.0 George Church in 2023 George ChurchGeneticist whose laboratory independently demonstrated CRISPR editing in human cells in 2013, and a founder of an unusually large number of genomics companies. Christopher Michel · CC BY-SA 4.0 Prof. Virginijus Šikšnys 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. Gie2016 · CC BY-SA 4.0

Últimas noticias sobre edición génica

Artículos reales de editores identificados. Cada titular enlaza al original: nunca los reescribimos ni republicamos.

Todas las noticias →
Backed by Amish and Mennonites, a new kind of gene therapy company takes shape LANCASTER, PA — This spring, Lorraine and Mark Martin, a Mennonite family with a tax business, adopted a stubby-legged cow named Royal Lite. Lorraine… Endpoints News Treatments 57 min ago↗ Rice plants can fight off devastating disease after gene editing with AI-designed tool University of Missouri researchers have created rice plants that resist bacterial blight, a devastating disease that threatens one of the world's most… Phys.org — Molecular Biology Agriculture 1h ago↗ STAT+: After two deaths in autoimmune cell therapy trial, Artiva is shutting down Recent deaths are setbacks in drug companies’ interest in applying cancer therapy strategies to autoimmune diseases. STAT Treatments 3h ago↗ Bonjour Genomics: A Video from ASHG GEN editors Uduak Thomas and Julianna LeMieux, PhD, recap ASHG, highlighting key sessions, Ultima Genomics’ user group meeting, Illumina news, and… GEN — Genetic Engineering News Companies 4h ago↗ Artiva to wind down after two patient deaths in autoimmune cell therapy trials Cell therapy biotech Artiva Biotherapeutics abruptly announced Tuesday morning that it is effectively shutting down and laying off almost all of its staff… Endpoints News Clinical Trials 7h ago↗ Outpace ends an ovarian cancer trial; Incyte data impresses at ESMO Plus news about Genmab, Rumi, Prana and Fractyl. Outpace Bio ends cell therapy trial: The Seattle startup canned a Phase 1 ovarian cancer trial of its… Endpoints News Clinical Trials 7h ago↗ STAT+: Nonprofit resurrects shelved gene therapy after startup shuts down Last year, U.K. gene therapy startup Bloomsbury Genetic Therapies announced it was shutting down after dosing just one patient. Now, a nonprofit is… STAT Treatments 7h ago↗ Spatial Proteomics for Precision Oncology In this GEN webinar, Bernd Bodenmiller, PhD (University of Zürich), illustrates this integrated approach through two recent examples. The post Spatial… GEN — Genetic Engineering News Companies 1d ago↗

CRISPR explicado

Empieza desde el principio. No se necesitan conocimientos de biología.

Empieza a aprender →
What is DNA?DNA is a long chemical chain that stores the instructions for building and running a living… What is a gene?A gene is a stretch of DNA that carries the instructions for making one particular thing the… What is a chromosome?A chromosome is one very long DNA molecule packaged with proteins; humans normally have 46, in… What is a genome?A genome is the complete set of genetic instructions in an organism: every chromosome, every… What is RNA?RNA is DNA's working copy: a temporary, single-stranded version the cell makes when it needs to… What is a mutation?A mutation is a change in DNA sequence. Most do nothing, some cause disease, and a few are…
An open journal, a magnifying glass and a molecular model under a desk lamp La investigación detrás Artículos clave resumidos en lenguaje claro, cada uno enlazado a la publicación original. A vintage microscope and a chain of glowing milestones La historia de la edición genética Desde la estructura del ADN hasta el primer medicamento CRISPR aprobado: los eventos que nos trajeron hasta aquí.
A farmhouse on a ploughed field at sunset AgriculturaCultivos, ganadería y las normativas que varían según el país Two empty chairs facing each other with a balance scale above ÉticaLos argumentos, presentados desde ambos lados A city skyline through a window with a glowing chart overlay InversiónCómo se financia esta industria: información, no asesoramiento The Earth at night with glowing connection lines Mapa mundialDónde ocurre la edición génica
Antes de seguir leyendo El CRISPR Decoded es una referencia educativa. No proporciona asesoramiento médico, y la información sobre una terapia experimental nunca es evidencia de que dicha terapia sea segura o eficaz. El estado de los ensayos clínicos y el regulatorio cambia con frecuencia. Si está en juego una decisión sobre tu propia salud o la de un familiar, habla con un profesional sanitario cualificado. Leer los avisos legales completos →