Category CRISPR

PIER MARIA FORNASARI
2025-12-07

Gene Therapy with exagamglogene autotemcel (exa-cel) Leads to Improved Quality of Life in Patients with Sickle Cell Disease and Beta Thalassemia

Substantial improvements seen across ages in overall health and wellbeing Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two studies published in Blood Advances. “This is the first time we’ve been able to measure improvements in quality of

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PIER MARIA FORNASARI
2022-11-10

New Exa-cel gene therapy for Sickle Cell Disease and Beta Thalassemia global regulatory submission in 2022

A potential 1-time gene editing treatment for severe sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) is entering approval review by the US Food and Drug Administration (FDA), the European Medicines Agency, and the UK Medicines and Healthcare products Regulatory Agency, according to a company statement. Both diseases involve variants in the gene encoding β globin. The variants

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PIER MARIA FORNASARI
2022-05-12

Novel Supramolecular CRISPR–Cas9 Carrier Enables More Efficient Genome Editing

Fifth generation polyrotaxane (PRX) carriers can effectively deliver CRISPR-Cas9 ribonucleoproteins (RNPs) The PRX carrier can cross the cell membrane, avoid endosomal action, and release Cas9 RNP for entry into the nucleusImage courtesy: Kumamoto University, The carriers, aminated polyrotaxanes, can flexibly and reversibly bind with Cas9 ribonucleoprotein and protect it from intracellular endosomal degradation CRISPR-Cas9 is

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PIER MARIA FORNASARI
2020-03-06

Scientists at Oregon edit genes inside the body using CRISPR for a hereditary blindness disorder, in a trial called BRILLIANCE.

A person with a genetic condition that causes blindness has become the first to receive a CRISPR–Cas9 gene therapy administered directly into their body. The treatment is part of a landmark clinical trial to test the ability of CRISPR–Cas9 gene-editing techniques to remove mutations that cause a rare condition called Leber’s congenital amaurosis 10 (LCA10).

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PIER MARIA FORNASARI
2020-01-03

Strategies for the CRISPR-Based Therapeutics

The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many

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PIER MARIA FORNASARI
2020-01-02

Will Prime Editors be the New CRISPR?

Early results suggest that prime editors are cleaner than CRISPR–Cas9 and more versatile than base editors, but many questions remain. A paper recently published in Nature from David Liu and co-workers discloses a ‘prime’ gene-editing system many years in the making. In principle, the system, comprising a catalytically impaired Cas9 enzyme and an engineered reverse transcriptase, may

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PIER MARIA FORNASARI
2019-10-29

Twitter reveals growing global public anxiety about Crispr gene-editing

The first ever analysis of all the tweets discussing Crispr gene-editing has revealed how the public feels about the technology. The findings suggest that over a six years public approval of gene-editing has cooled and that most people chatting about the technology are worried. Twitter offers researchers a vast amount of semi-structured data and provides direct access

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