La guida definitiva all'editing genico.
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La mappa della rivoluzione dell'editing genico

Il CRISPR Decoded

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.

15 treatments tracked 14 clinical trials Riferimento educativo · solo per ricerca · non costituisce consulenza medica
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.

Inizia da dove ti trovi davvero

Questo sito è scritto per lettori molto diversi tra loro. Ogni porta qui sotto porta nello stesso database da una prospettiva diversa. Niente è semplificato in modo eccessivo, e niente presuppone una laurea in biologia.

A child's hands holding a small glowing DNA ladder New to all of thisParti da cos'è il DNA e costruisci passo dopo passo. Non serve alcuna preparazione di base. A doctor and a patient talking across a desk A patient or familyCosa è approvato, cosa è ancora in sperimentazione e come distinguere le due cose. Nested glowing rectangles receding into darkness Seguire il settoreChi sta sviluppando cosa, a quale fase. Informazioni, mai consigli medici. Rows of seedlings under purple grow lights Qui per cibo e agricolturaColture e animali modificati geneticamente, e perché le regole variano da paese a paese.

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.

Tutte le tecnologie →
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 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.

Tutte le malattie →
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

Trattamenti

Farmaci con nome proprio, con lo stato regolatorio indicato in ogni scheda.

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

Farmaci approvati in CRISPR Decoded: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)

Tutti i trattamenti →

Sperimentazioni cliniche

Dove vengono prodotte davvero le prove scientifiche.

4In fase di reclutamento
2Attivo, non in reclutamento
7Completata
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

Cerca nel database degli studi →

Aziende da conoscere

Chi sta costruendo cosa e a che punto è ciascun programma.

Tutte le aziende →
CRISPR TherapeuticsCRSP · Zug, Switzerland Intellia TherapeuticsNTLA · Cambridge, Massachusetts Beam TherapeuticsBEAM · Cambridge, Massachusetts Editas MedicineEDIT · Cambridge, Massachusetts Prime MedicinePRME · Cambridge, Massachusetts Verve TherapeuticsPrivato · Boston, Massachusetts Caribou BiosciencesCRBU · Berkeley, California Sana BiotechnologySANA · Seattle, Washington

Scienziati che hanno cambiato il campo

Cosa ha realmente contribuito ogni persona. L'attribuzione dei meriti in questo campo è controversa, e noi lo diciamo chiaramente.

Tutti gli scienziati →
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

Ultime notizie sul gene editing

Articoli reali da editori identificati. Ogni titolo rimanda all'originale. Non li riscriviamo né li ripubblichiamo mai.

Tutte le notizie →
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 56 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 spiegato

Inizia dall'inizio. Non è necessaria una preparazione in biologia.

Inizia a imparare →
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 ricerca che c'è dietro Articoli fondamentali riassunti in linguaggio semplice, ciascuno collegato alla pubblicazione originale. A vintage microscope and a chain of glowing milestones La storia dell'editing genico Dalla struttura del DNA alla prima medicina CRISPR approvata: gli eventi che ci hanno portato fin qui.
A farmhouse on a ploughed field at sunset AgricolturaColture, bestiame e normative che variano da paese a paese Two empty chairs facing each other with a balance scale above EticaGli argomenti, presentati da entrambe le parti A city skyline through a window with a glowing chart overlay InvestimentiCome si finanzia questo settore: informazioni, non consulenza The Earth at night with glowing connection lines Mappa del mondoDove avviene l'editing genico
Prima di continuare a leggere Il CRISPR Decoded è una risorsa educativa. Non fornisce consulenza medica, e le informazioni su una terapia sperimentale non costituiscono mai una prova che essa sia sicura o efficace. Lo stato delle sperimentazioni cliniche e delle approvazioni regolatorie cambia frequentemente. Se devi prendere una decisione riguardo alla tua salute o a quella di un familiare, rivolgiti a un professionista sanitario qualificato. Leggi le note legali complete →