Карта революции в редактировании генов
CRISPR Decoded
Исследуйте технологии, методы лечения, болезни, учёных, компании, клинические исследования и открытия, которые формируют будущее генетической медицины.
15 treatments tracked
14 clinical trials
Справочная информация в образовательных целях · только для исследований · не является медицинским советом
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Начните с того места, где вы находитесь
Этот сайт написан сразу для очень разных читателей. Каждая дверь ниже ведёт в одну и ту же базу данных, но под разным углом. Здесь ничего не упрощено, и ничто не предполагает диплома биолога.
New to all of thisНачните с самого понятия ДНК и двигайтесь дальше. Никакой предварительной подготовки не нужно.
A patient or familyЧто одобрено, что только проходит испытания и как отличить одно от другого.
Следим за отрасльюКто и что разрабатывает, на каком этапе. Информация, а не советы.
Интересуетесь едой и сельским хозяйствомГенно-редактированные культуры и сельскохозяйственные животные, а также различия в правилах регулирования по странам.
Изучить редактирование генов
Семь способов, которыми учёные изменяют генетический материал. Каждая страница начинается с доступного объяснения, а затем углубляется настолько, насколько вам нужно.
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
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.
Сравнить их бок о бокТочность, разрезание, размер полезной нагрузки, доставка, зрелость, риски
Заболевания, которые являются мишенями
На каждой странице заболевания чётко указано, является ли редактирование генов одобренным методом лечения, проходит ли испытания или пока остаётся лишь лабораторной идеей.
Sickle Cell DiseaseBlood disorders
SpicyMilkBoy · CC BY-SA 4.0
Beta ThalassemiaBlood disorders
Dr Graham Beards · CC BY-SA 4.0
ATTR AmyloidosisMetabolic disease
Hereditary AngioedemaRare genetic disease
LucyHAE · CC BY-SA 3.0
High Cholesterol and Familial HypercholesterolemiaCardiovascular disease
CancerCancer
Mikael Häggström, M.D. Author info... · CC0
Leukemia and LymphomaCancer
https://wellcomeimages.org/indexplu... · CC BY 4.0
HIVInfectious disease
Photo Credit: C. Goldsmith
Content... · Public domain
Duchenne Muscular DystrophyMuscular disease
Dr. Edwin P. Ewing, Jr. · Public domain
Cystic FibrosisRare genetic disease
Методы лечения
Конкретные препараты с указанием регуляторного статуса в каждой карточке.
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.
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.
Zynteglo (betibeglogene autotemcel)An approved gene-addition therapy for transfusion-dependent beta thalassemia: the non-editing alternative to Casgevy in the same disease.
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.
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.
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.
Одобренные лекарства в CRISPR Decoded: Casgevy (exagamglogene autotemcel) · Lyfgenia (lovotibeglogene autotemcel) · Zynteglo (betibeglogene autotemcel)
Клинические исследования
Где в действительности формируется доказательная база.
4Идёт набор участников
2Активно, набор завершён
7Завершено
CLIMB SCD-121: the trial that led to the first CRISPR medicine approvalSickle cell disease · NCT03745287
CLIMB THAL-111: the beta thalassemia trial behind the Casgevy approvalBeta thalassemia · NCT03655678
MAGNITUDE: Phase 3 in vivo CRISPR for ATTR cardiomyopathyATTR amyloidosis · NCT06128629
MAGNITUDE-2: Phase 3 in vivo CRISPR for ATTR polyneuropathyATTR amyloidosis · NCT06672237
HAELO: the first positive Phase 3 result for in vivo gene editingHereditary angioedema · NCT06634420
BEACON: base editing for sickle cell diseaseSickle cell disease · NCT05456880
Компании, которые стоит знать
Кто что разрабатывает и на каком этапе находится каждая программа.
CRISPR TherapeuticsCRSP · Zug, Switzerland
Intellia TherapeuticsNTLA · Cambridge, Massachusetts
Beam TherapeuticsBEAM · Cambridge, Massachusetts
Editas MedicineEDIT · Cambridge, Massachusetts
Prime MedicinePRME · Cambridge, Massachusetts
Verve TherapeuticsЧастный · Boston, Massachusetts
Caribou BiosciencesCRBU · Berkeley, California
Sana BiotechnologySANA · Seattle, Washington
Учёные, изменившие область науки
Чей именно вклад и в чём, авторство в этой области оспаривается, и мы указываем, где именно.
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
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
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 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 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
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
Последние новости о редактировании генов
Настоящие статьи от известных издателей. Каждый заголовок ведёт на оригинал, мы никогда не переписываем и не перепечатываем материалы.
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STAT+: After two deaths in autoimmune cell therapy trial, Artiva is shutting down
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Bonjour Genomics: A Video from ASHG
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Outpace ends an ovarian cancer trial; Incyte data impresses at ESMO
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STAT+: Nonprofit resurrects shelved gene therapy after startup shuts down
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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…
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…
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.
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…
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…
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…
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…
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…
CRISPR простыми словами
Начните с самого начала. Знания биологии не нужны.
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…
Прежде чем читать дальше
CRISPR Decoded, образовательный справочник. Он не даёт медицинских рекомендаций, а информация об экспериментальной терапии ни в коем случае не является доказательством её безопасности или эффективности. Статус клинических исследований и регуляторный статус часто меняются. Если вы принимаете решение, касающееся вашего здоровья или здоровья члена вашей семьи, проконсультируйтесь с квалифицированным медицинским специалистом.
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