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"Nature": A new breakthrough in gene therapy turns the mutant protein into a healthy protein

 Last Update: 2021-06-08
 Source: Internet
 Author: User

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Today, the latest batch of "Nature" papers went online as scheduled.

This study is aimed at sickle cell disease (SCD).

No matter which method is used, good results have been achieved in clinical trials, and a functional cure for SCD can be achieved.

This paper provides a brand new idea.

From a rare hemoglobin variant in Indonesia, researchers have found new ideas.

In human cells, the researchers conducted a proof-of-concept.

▲Single-base editing of mouse hematopoietic stem cells can reverse their symptoms (picture source: reference [1])

In another experiment, the researchers isolated hematopoietic stem and progenitor cells from a humanized SCD mouse model, performed single-base editing, and then fed these cells into irradiated mice.

Note: The original text has been deleted

Reference materials:

[1] Newby, GA, Yen, JS, Woodard, KJ et al.

[2] Relying on an ultra-rare variant, David Liu unveils a new approach to editing sickle cell, Retrieved June 2, 2021, from https://endpts.
com/relying-on-an-obscure-variant-david-liu -unveils-a-new-approach-to-editing-sickle-cell/

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