Research and Development

       There are around 56000 newborns suffering from severe thalassemia in China each year, and of which at least 30000 patients have to be treated with blood transfusion. Even worse, around 5500 cases of ɑ-thalassemia dead before birth each year. South China is the high incidence area of thalassemia, which the genetic defects rate is around 2.5 % to 20 %. Especially in Guangdong and Guangxi province, the genetic defects rate is as high as 10 %~20 %.
       Thalassemia is a kind of hereditary anemia caused by globin gene deficiency. At present, there are at least 81 and 186 types of mutation for ɑ and β thalassemia, respectively. The traditional therapies for thalassemia are long-term blood transfusion and stem cell transplantation (allograft), while both of them show serious side-effect and immune rejection. Gene therapy, which directly edits the mutant sites in patients’ stem cells, is a promising treatment for thalassemia and sickle cell disease.
       For our thalassemia program, gene editing was employed as core technology for thalassemia therapy. The key mutant site targeted editing by CRISPR was corrected.

病毒种类

质粒大小

适合菌株

pX330

8506 bp

TOP10

pX458

9288 bp

TOP10

lentiCas9nD10A-blast

12860 bp

TOP10

pX330A-lx2

8964 bp

TOP10

pX330A-Fok I-lx3

9476 bp

TOP10

pY010(pcDNA3.1-hAsCpf1)

9464 bp

TOP10

pX461

9289 bp

TOP10

pX601

7446 bp

TOP10

NL4.3-BaL

14828 bp

HB101

pCMV-VSV-G

6.507 kb

HB101

pUC19

2.686 kb

TOP10

psPAX2

10.668 kb

Stbl3

ssAAV-aBR1-CMV-EGFP-aBL3

5.393 kb

HB101

pCAR-hCD19-GMCSF-EGFRt

1.021 kb

HB101

pHelper(AAV6)

11.635 kb

Stbl3

pAAV-RC6(AAV6)

7.33 kb

Stbl3

pAAV-GFP

5.378 kb

HB101

@ 2017 Guangdong Trement Medical Science and Technology Co., Ltd