Application and prospect of gene therapy for fundus vascular diseases / 国际眼科杂志(Guoji Yanke Zazhi)

Fundus vascular diseases, including neovascular age-related macular degeneration(nAMD)and diabetic retinopathy(DR), are the leading causes of visual impairment worldwide. With the accelerated aging and increased incidence of diabetes, the prevalence of these two fundus diseases will continue to rise. Currently, intraocular injection of anti-vascular endothelial growth factor(anti-VEGF)remains the first-line treatment for fundus vascular diseases, but disadvantages exist, such as frequent intraocular injections, high cost and poor compliance, thus more durable and effective therapeutic strategies need to be explored. The successful application of gene therapy in inherited retinal diseases(IRDs)provides a new idea for the treatment of fundus vascular diseases. With the ongoing of several clinical trials, gene therapy for fundus vascular diseases is expected to be employed in the clinical setting. But there still remain some concerns, including the optimal therapeutic targets selection, administration route and safety issues. This review focuses on the application and prospect of gene augmentation and gene editing-mediated anti-VEGF therapy for the treatment of nAMD and DR.

Treatment of leukemia by allogeneic peripheral blood stem cell transplantation from sibling donors / 中国实验血液学杂志

This study was aimed to observe the efficacy and side effects of allogeneic peripheral blood stem cell transplantation (Allo-PBSCT) in patients with leukemia. The donors were siblings matched with t HLA-A, B, DR loci of recipient. After mobilization with 250 microg/day rhG-CSF for 5 days, peripheral blood stem cells were collected 1 to 2 times. 4 patients with leukemia received 6.78 x 10(8)/kg +/- 1.96 x 10(8)/kg (5 x 10(8)/kg-8.67 x 10(8)/kg) peripheral blood mononuclear cells, which contained 15.02 x 10(6)/kg +/- 8.93 x 10(6)/kg (5.3 x 10(6)/kg-24.23 x 10(6)/kg) CD34(+) cells after modified Bu/Cy conditioning regimen and MTX + CsA + MMF were given for prophylaxis of aGVHD. The results showed that the leukocyte was reduced to the lowest at pretransplantation 2 days-posttransplantation 2 days. Neutrophil amount >0.5 x 10(9)/L was found at 11 - 17 day after transplantation, platelet >50 x 10(9)/L-at 11 - 55 days after transplantation. Out of 4 patients, aGVHD, cGVHD and infection took place in 2,2 and 2 cases, respectively. Bone marrow displayed hematopoietic recovery at 28 day after transplantation, examination of STR in DNA revealed proliferation of donor cells in patients. In conclusion, Allo-PBSCT in combination with modified Bu/Cy conditioning regimen and MTX + CsA + MMF prophylaxis of aGVHD is safety and reliable for treatment of leukemia.