Description: Autologous, ex vivo CRISPR/Cas9 gene-edited therapy in which aims to edit a patient's own hematopoietic stem cells to produce fetal hemoglobin ...

We have established a portfolio of programs by selecting disease targets based on a number of criteria, including unmet medical need, technical feasibility, ...

17 янв. 2024 г. · The FDA and MHRA approval of Casgevy marks a pinnacle moment for the industry as the first CRISPR therapy to hit the market.

13 мар. 2024 г. · CRISPR clinical trials 2024: A comprehensive update on gene-editing clinical trials and the landscape following the first approved therapy.

The CRISPR-del pipeline effectively generates a nearly complete, bi-allelic deletion of a large protein-coding gene. For the purpose of achieving complete gene ...

We provide guidelines to identify CRISPR targets sites most likely to cause loss of protein function, two protocols to generate gRNA,2,3 and a pipeline to ...

5 янв. 2024 г. · The Massachusetts-headquartered company announced its 2024 aim to complete patient enrolment for studies evaluating NTLA-2001 and NTLA-2002.

We are researching and developing two types of gene editing medicines: in vivo gene editing medicines that edit genes inside the body, and ex vivo gene editing ...

We have used this pipeline to create mutations in 13 genes to date and provide an example of the pipeline using six1a CRISPR.

17 янв. 2019 г. · Here, we report an improvement of the GUIDE-seq method, iGUIDE, which allows filtering of mispriming events to clarify the true cleavage signal.