El bebé “arreglado a medida”: la edición genética que abre una nueva era médica

Un recién nacido con una enfermedad metabólica letal recibió la primera terapia CRISPR personalizada del mundo, diseñada solo para él. Los resultados iniciales muestran que su organismo empezó a funcionar como si la mutación nunca hubiera existido. Por Alina C. Galifante para NLI Un caso que cambia la historia de la medicina El protagonista es un bebé con una mutación gravísima en el gen CPS1, responsable de un trastorno metabólico que impide procesar adecuadamente el amoníaco. […]

https://noticiaslainsuperable.com.ar/2025/12/09/el-bebe-arreglado-a-medida-la-edicion-genetica-que-abre-una-nueva-era-medica/

What is the therapeutic potential of correcting mutations by base editing of the #MitochondrialGenome? This study shows that #mitochondrial #BaseEditing can indeed functionally create & correct mitochondrial pathogenic #mutations in patient-derived cells @PLOSBiology https://plos.io/3ZMa2K2

holy moly! a baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for them:

https://www.cbc.ca/news/health/gene-therapy-baby-pennsylvania-1.7536105

#CRISPR #BaseEditing

Baby with rare disease called first to successfully receive personalized gene therapy | CBC News

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him.

CBC
Repairing a deleterious domestication variant in a floral regulator gene of tomato by base editing.
#GenomeEditing #Tomato #BaseEditing #CropImprovement
https://www.nature.com/articles/s41588-024-02026-9
Repairing a deleterious domestication variant in a floral regulator gene of tomato by base editing - Nature Genetics

A deleterious mutation in the tomato transcription factor SSP2 was enriched during domestication. Repairing the deleterious mutation in cultivated tomato by base editing leads to compact growth and early fruit yield.

Nature

What's next in #plant #gene editing?

Our latest #JIPB Brief Comms feature highlights work by Liu et al. who are developing #guanine base editors for G-to-T editing in #rice.

https://doi.org/10.1111/jipb.13729
@wileyplantsci
#PlantSci #BaseEditing #CRISPR #Cas9 #CropSci #botany

Looking to perform genome-wide screens in bacteria? 🦠 Look no further than base editors—with a few tweaks #HIRI’s Beisel lab introduced to enhance the number of targetable sites. #baseediting #CRISPR #Cas9
https://doi.org/10.1093/nar/gkae174
Expanding the flexibility of base editing for high-throughput genetic screens in bacteria

Abstract. Genome-wide screens have become powerful tools for elucidating genotype-to-phenotype relationships in bacteria. Of the varying techniques to achieve k

OUP Academic

Base editing, a new form of gene therapy, sharply lowers bad cholesterol in clinical trial

A technique for precisely rewriting the genetic code directly in the body has slashed “bad” cholesterol levels—possibly for life—in three people

“It worked. But we won’t know for years how safe this is"

#GeneTherapy #BaseEditing #MedMastodon
https://www.science.org/content/article/base-editing-a-new-form-of-gene-therapy-sharply-lowers-bad-cholesterol

3rd ARRIGE Albrecht Müller Seminar (free online conference) on ‘Human Therapeutic Applications of Gene Editing’ November 13th, 2023 – 2pm-5pm (CET) – Paris time.
#CRISPR #GenomeEditing #ClinicalTrials #BaseEditing #primeediting #crisprcas9 #genetherapy #advancedtherapies #ARRIGE #ResponsibleResearch #ethics #bioethics #rri #innovation
Full information and registrarion link to gain free access to this meeting available at:
https://www.arrige.org/portfolio/3rd-albrecht-muller-arrige-seminar/
3rd Albrecht Müller ARRIGE Seminar – ARRIGE

Pharma Company Gains Precise Gene Edits in $2B Biotech Deal

A global drug maker is acquiring access to a #biotechnology company's precise #genome editing techniques to develop new #therapies for rare and cardiometabolic disorders.

https://sciencebusiness.technewslit.com/?p=44824

#News #Science #Business #Genomics #GeneEditing #Crispr #Enzymes #Chemistry #BasePairs #BaseEditing #Collaboration #Licensing #RareDiseases

Pharma Company Gains Precise Gene Edits in $2B Biotech Deal

A global drug maker is acquiring access to a biotechnology company's precise genome editing techniques to develop new therapies for rare and cardiometabolic disorders.

Science and Enterprise

What's this sound?

This? This is just evoCDA1 editing the Cs at the target site of ALL the cells of this little fly.

#Drosophila #CRISPR #geneediting #baseediting