Application of Gene Therapy in Hemophilia.

Gene therapy refers to introducing normal exogenous genes into target cells to correct or compensate for the diseases caused by defective and abnormal genes for the purpose of therapy. It holds out hope of a cure for single-gene genetic diseases such as thalassemia, hemophilia, etc. At present, gene therapy is performed in two ways: introducing exogenous genes, and gene editing. A great number of clinical trials of gene therapy in hemophilia have been carried out using viral vectors to introduce foreign genes into target cells. However, the production of neutralizing antibodies following injection and the inability to prepare viral vectors in large quantities limit their application. Although gene-editing methods like CRISPR avoid the above problems, the potential risks of off-target effects are still unknown. More trials and evidence are needed to elucidate the safety and accuracy of gene therapy. This paper will review the bench and clinical work of gene therapy in hemophilia in recent years, and summarize the challenges and prospects of gene therapy, so as to provide directions for future scientific research in this field.

The Limitation of Randomized Control Trials on the Influence of Acupuncture and In Vitro Fertilization: A Literature Review.

Objective: Acupuncture has gained popularity among patients undergoing in vitro fertilization (IVF). However, the beneficial effect of acupuncture for improving IVF success is controversial and debatable. Given that different meta-analyses have come to different conclusions, it is crucial to explore the clinical trials in more detail. This literature review examined the limitation of randomized controlled trials (RCTs) on the influence of acupuncture in IVF. Methods: This review began with 844 studies. The inclusion criteria were studies that had acupuncture treatments in conjunction with IVF. After exclusion criteria were applied, the final number of peer-reviewed studies was 10. Results: There were substantial variations in the results of the 10 RCTs. This seemed to suggest that acupuncture was not effective in conjunction with IVF treatment. However, limitations emerged that might explain these variations in results. Such limitations include timing of acupuncture and point selections; acupuncture not performed by experienced licensed acupuncturists; lack of Traditional Chinese Medicine diagnoses and fixed protocols causing biases; acupuncture dosages; and using sham acupuncture as a control. Conclusions: There is an urgent need for further research into the effectiveness of acupuncture for improving IVF outcomes. This review provides insight into this complex and controversial topic, revealing limitations of the clinical trials that led to different conclusions. If future research can examine acupuncture treatment carefully to resemble real-world clinical practice-having appropriate controls and individualized acupuncture treatments-increasingly positive effects from acupuncture in IVF may be expected.

Global burden and attributable risk factors of acute lymphoblastic leukemia in 204 countries and territories in 1990-2019: Estimation based on Global Burden of Disease Study 2019.

To provide a foundational guideline for policy-makers to efficiently allocate medical resources in the context of population aging and growth, the latest spatial distribution and temporal trend of acute lymphoblastic leukemia (ALL) along with attributable risk factors by sex and age were mapped. Based on the Global Burden of Disease Study 2019, estimated annual percentage change (EAPC) was calculated according to the relativity between age-standardized rate and calendar year, to quantify temporal trends in morbidity and mortality of ALL. We used applied Spearman rank correlation to estimate the relationship between the EAPC and potential influence factors. The population attributable fraction of potential risk factors for ALL-related disability-adjusted life years were estimated by the comparative risk assessment framework. As a result, we found that new ALL cases increased significantly by 1.29% worldwide, and the age-standardized incidence rate increased by 1.61% annually. The proportion of elder patients sharply increased, especially within the higher socio-demographic index (SDI) region. Smoking and high body mass index remained the predominant risk factors for ALL-related mortality. Notably, the contribution of high body mass index presented an increasing trend. In conclusion, the global burden of ALL has steadily increased, especially in Middle SDI region. Health measures and new drugs should be taken into consideration to improve the management and treatment of elders with ALL due to an increasing proportion in the higher SDI region. For Low SDI areas, attention should be paid to the environmental problems caused by industrial development.

Magnitude and Temporal Trend of the Chronic Myeloid Leukemia: On the Basis of the Global Burden of Disease Study 2019.

PURPOSE: To map the magnitudes and temporal trends of chronic myeloid leukemia (CML) along with its attributable risk factors, providing the essential foundation for targeted public policies at the national, regional, and global levels. MATERIALS AND METHODS: We retrieved annual data on CML burden in 204 countries and regions from the Global Burden of Disease Study 2019 in 1990-2019. The estimated annual percentage change (EAPC) was calculated to quantify the temporal trends of CML burden by region, sex, and age group. RESULTS: Globally, the age-standardized incidence rate of CML declined weakly over the past few years (EAPC: -1.04), but the number of incident cases increased by 54.1% to 65.8 × 103 in 2019. By contrast, a dramatic drop in death and disability-adjusted life years (DALYs) rate (EAPCs: -2.55; -2.69) led to a reduction in deaths and DALYs, especially in high-income regions. In 2019, the highest age-standardized death rate was observed in Ethiopia (1.89 per 100,000). The death rate of CML was pronounced among the population age above 70 years. DALYs of CML worldwide were primarily attributable to smoking (12.2%), high body mass index (5.0%), occupational exposure to benzene (0.9%), and occupational exposure to formaldehyde (0.3%) in 2019. CONCLUSION: Although the mortality rate of CML has decreased significantly, the management of patients with CML cannot be neglected, especially in elders and developing regions.