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遺伝学が初めてですか？　閲覧しながら、すべての用語をわかりやすく説明します。同じページに、サポートが組み込まれています。

専門家の見解
生物学はすでにご存知の方へ。コンテンツだけをすっきりとコンパクトに、余分な説明なしでお届けします。これがデフォルト表示です。

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A lone figure before a wall of streaming light

ゲノム編集革命の地図

CRISPR Decoded

遺伝子医療の未来を形づくる技術・治療・疾患・科学者・企業・臨床試験・発見を探ってみましょう。

15 treatments tracked 14 clinical trials 教育的参考情報・研究目的のみ・医療アドバイスではありません
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.

今いる場所から始める

このサイトはまったく異なる読者を同時に対象として書かれています。以下の入口はそれぞれ異なる角度から同じデータベースにつながっています。内容を簡略化することも、生物学の学位を前提とすることもありません。

A child's hands holding a small glowing DNA ladder New to all of thisDNAとは何かから始めて、順を追って学べます。事前知識は不要です。 A doctor and a patient talking across a desk A patient or family承認済みのもの、試験段階のもの、そしてその見分け方。 Nested glowing rectangles receding into darkness 業界の動向を追う誰がどの段階で何を開発しているか。情報の提供であり、医療アドバイスではありません。 Rows of seedlings under purple grow lights 食と農業について知りたい方へ遺伝子編集された農作物と家畜、そして国によってルールが異なる理由。

遺伝子編集を探索する

科学者が遺伝物質を変える7つの方法。各ページはわかりやすい説明から始まり、知りたい深さまで掘り下げられます。

技術一覧 →
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 並べて比較する精度・切断・カーゴサイズ・デリバリー・成熟度・リスク

対象疾患

各疾患ページには、遺伝子編集が承認済みの治療法なのか、臨床試験中なのか、まだ研究段階にすぎないのかが、わかりやすく記載されています。

疾患一覧 →
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

治療法

すべての記録に規制状況が記載された、名称付きの医薬品。

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

CRISPR Decodedの承認済み医薬品: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)

治療法一覧 →

臨床試験

エビデンスが実際に生まれる場所。

4募集中
2募集終了・試験継続中
7完了
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

試験データベースを検索 →

注目の企業

誰が何を開発しているか、そして各プログラムがどこまで進んでいるか。

企業一覧 →
CRISPR TherapeuticsCRSP · Zug, Switzerland Intellia TherapeuticsNTLA · Cambridge, Massachusetts Beam TherapeuticsBEAM · Cambridge, Massachusetts Editas MedicineEDIT · Cambridge, Massachusetts Prime MedicinePRME · Cambridge, Massachusetts Verve Therapeutics非公開 · Boston, Massachusetts Caribou BiosciencesCRBU · Berkeley, California Sana BiotechnologySANA · Seattle, Washington

この分野を変えた科学者たち

それぞれの人が実際に何に貢献したか――この分野では功績の帰属が争われており、その箇所を明示しています。

科学者一覧 →
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

遺伝子編集の最新ニュース

実名の出版社による本物の記事です。すべての見出しは元の記事にリンクしており、私たちが書き直したり転載したりすることはありません。

ニュース一覧 →
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とは

最初の一歩から始めましょう。生物学の知識は必要ありません。

学習を始める →
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 その背景にある研究 主要な論文をわかりやすい言葉で要約し、それぞれ元の論文にリンクしています。 A vintage microscope and a chain of glowing milestones ゲノム編集の歴史 DNAの構造から最初に承認されたCRISPR医薬品まで——現在に至るまでの出来事をたどります。
A farmhouse on a ploughed field at sunset 農業農作物・家畜と国ごとに異なるルール Two empty chairs facing each other with a balance scale above 倫理両方の立場からの意見 A city skyline through a window with a glowing chart overlay 投資この業界はどのように資金調達しているか――情報提供であり、投資アドバイスではありません The Earth at night with glowing connection lines 世界地図遺伝子編集が行われる場所
続きを読む前に CRISPR Decodedは教育目的のリファレンスです。医療アドバイスを提供するものではなく、実験的療法に関する情報は、その療法の安全性や有効性を示す根拠にはなりません。臨床試験や規制上のステータスは頻繁に変わります。ご自身やご家族の健康に関わる判断が必要な場合は、資格を持つ医療専門家にご相談ください。 免責事項の全文を読む →