NEJM: Using CRISPR-Cas9 for in-vivo gene editing to treat transthyretin amyloidosis

On June 26, 2021, NEJM, the world's top medical journal, published that the CRISPR in vivo genome editing therapy NTLA-2001 achieved positive results in a phase 1 clinical trial
.
Among the 6 patients receiving treatment, 3 patients received a dose of 0.

1 mg/kg and 3 patients received a dose of 0.

On June 26, 2021, NEJM, the world's top medical journal, published that the CRISPR in vivo genome editing therapy NTLA-2001 achieved positive results in a phase 1 clinical trial

Transthyretin amyloidosis (ATTR) is a serious and rare genetic disease that is life-threatening.

It is estimated that there are about 50,000 patients worldwide.

NTLA-2001 Nobel Prize in Chemistry for Therapy 2020

The CRISPR gene editing system, known as the "Magic Scissor", can specifically edit almost any sequence in the genome
.
However, one of the key issues that need to be solved when this system is used to treat human diseases is its safety-"off-target effect", that is, if the CRISPR system edits a DNA sequence that should not be edited in the body, it may introduce genetic mutations.

, Resulting in undesirable consequences such as cell canceration

In this phase 1 clinical trial, Intellia researchers injected a new CRISPR therapy, NTLA-2001, into the blood of 6 patients with transthyretin amyloidosis, which is a rare and uncommon Fatal genetic disease

.

In this phase 1 clinical trial, Intellia researchers injected a new CRISPR therapy, NTLA-2001, into the blood of 6 patients with transthyretin amyloidosis, which is a rare and uncommon Fatal genetic disease

The liver of patients with transthyretin amyloidosis produces a destructive protein, and CRISPR therapy is designed to target the gene that produces this protein and prevent it from working

After conducting in vitro and in vivo preclinical studies, we evaluated the safety and pharmacodynamic effects of a single escalating dose of NTLA-2001 in 6 patients with hereditary ATTR amyloidosis with polyneuropathy, and two initial dose groups There are 3 (0.

Preclinical studies have shown that TTR is permanently knocked out after a single dose

CRISPR therapy caused only minor side effects and did reduce the level of the destructive protein.

If it goes well, NTLA-2001 will provide a better treatment option for transthyretin amyloidosis.

More exciting than NTLA-2001 for the treatment of transthyretin amyloidosis is that CRISPR injection therapy can now be considered to treat other genetic diseases that are difficult to directly target, such as heart disease or brain diseases

Original source:

Original source: Original source:

Gillmore et al.
, (2021).
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
The New England Journal of Medicine,DOI: 10.
1056/NEJMoa2107454

Gillmore et al.
, (2021).
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
The New England Journal of Medicine,DOI: 10.
1056/NEJMoa2107454 Gillmore et al.
, (2021).
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
The New England Journal of Medicine,DOI: 10.
1056/NEJMoa2107454 Gillmore et al.
, (2021).
CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.
The New England Journal of Medicine,DOI: 10.
1056/NEJMoa2107454CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis.Leave a message here