Gaucher disease is a commonly inherited, autosomtic recessive lysosomal storage disorder, due to the mutation of structure gene encoding beta-glucocerebrosidase (GC) resulted in the deficiency of the enzyme. Besides enzyme replacement, there is no good ways to treat it. This review briefly introduced the phenotypes, pathologic physiology and general therapy to the disease, strongly stress the genetherapy. From animals to human, from in vitro to in vivo, the review detaily descibe the rapid progress made in genetherapy of Gaucher disease.

Drug clinical trial is an important link in the chain of new drug research and development. The results of drug discovery and development directly depend on the extent of standardization of clinical trials. Therefore, improving the quality of drug clinical trials is of great importance, and drug clinical trial institutions play a crucial role in the quality management of drug clinical trials. After years of development, the overall level of drug clinical trials has advanced rapidly in China, and a large number of clinical trials of traditional Chinese medicine have also been carried out. However, there is still a big gap between our country and developed countries. Therefore, for the construction and management of Chinese drug clinical trial institutions, there is still a long way to go. This study aims to analyze the current development of drug clinical trial institutions in China and explore the existing problems from three aspects, including current situations of institutional organization and management, regional and professional distributions, and quality control. And some suggestions are put forward finally, including support of traditional Chinese medicine, introduction of drug-risk management system, and construction of information management.
China ; Clinical Trials as Topic ; standards ; Drug Evaluation ; Drug Therapy ; standards ; trends ; Drugs, Chinese Herbal ; standards ; therapeutic use ; Humans ; Quality Control ; Research

About 50％ to 60％ of the stage Ⅱ-Ⅲ colorectal carcinoma patient suffer from liver metastases.Recently,there is a research hotspot on how to improve the prognosis of the colorectal carcinoma liver metastases (CRLM) patient.It can elucidate the way how the carcinoma cells transfer from the original tumor to the liver,through establishing a CRLM animal model.An ideal CRLM animal model should perfectly mimic the total procedure of the metastases pathway,including the change of the cytology behavior and the development of the tumor biology.Besides,the model should be practical,tumor-predictable and accord with the ethical consideration.Until now,there is not an ideal CRLM animal model can perfectly match those conditions.This review goes through the advantages and disadvantages of different CRLM animal model methods used for research.

Objective To establish a method for determination of Icariin in Yishengling granules (Sucrose-free) by HPLC. Methods Using a column packed with C18 and mixture of acetonitrile-water (30∶70) as the mobile phase. The flow rate was 1.0 mL/min and the temperature was 30 ℃. The detection wavelength was at 270 nm. Result The linear range of Icariin was 4.1～20.5 ?g (r=1). The mean recovery was 102.90% and RSD was 1.72%. Conclusion The method is simple, reliable and rapid, and can be applied to the quality control of Yishenling granules (Sucrose-free).

Objective To develop a new ambulance aid on airport rescue of pilot and solve the difficult problems on medical staff, which carry the wounded from high aircraft cabin to the ground. Methods The device owned QG 0.85 trailer chassis. On the basis of it, self-made manual flexible extension-jib, flexible extension ladders and safety board were designed. The safety board was used to fix wounded pilots to protect secondary injury of backbone and C-spine. Results The maximal speed of equipment was 80km/h and spreading time is 3～7min, operating time is 4min and lifting height is 6 000mm. Conclusion The ambulance aid can be used in the location of flight accident quickly and rescued the pilots rapidly, adapted to any type fighters and met the needs of medical support in flight.

Objective To learn the current deployment and problems of medical equipment at 44 hospitals directly under or managed by the NHFPC,studying the correlation between the number of equipment deployment,and amount of outpatients/inpatients and inpatient surgeries for recommendations on scientific and rational deployment of medical equipment.Methods Literature review,expert consultation and questionnaire survey methods were used to study the use and problems in hospital equipment deployment.EpiData was used to build a database,and the means,standard variations and constituent ratios were applied for descriptive analysis of medical equipment deployment of the hospital departments.The data of hospital equipment usage and staffing,hospital beds,and service volume were subject to correlation analysis.Results 24 of the 44 hospitals were deployed with class-A medical equipment,covering eight types and 43 devices;the 44 hospitals were deployed with 441 class-B equipment of five types;the equipment types in great demand at such hospitals were MRI (77.27%),CT (70.45%),bioanalysis devices (68.18%),conventional radiation devices (52.27%),and conventional equipments (54.55%).The survey found a positive correlation ( P <0.05 ) between the equipments,and the amount of outpatients/inpatients and inpatient surgeries.Conclusions Hospitals should further establish and improve their deployment management system,rationalize their deployment procedures,and use scientific and rational deployment.Such approaches as equipment sharing,centralized management and performance appraisal could be called into play to improve equipment efficiency.

Objective To observe the inhibition of vascular endotheilal growth factor (VEGF) gene transcribition and translation with antisense VEGF165 adenovirus vector transfection. Method To transfect the synovial cells with antisense VEGF165 adenovirus and detect the gene translation level with northern-blot method. Result The Northern-blot showed that: the transcription of VEGF gene were inhibited markedly after the antisense VEGF165 gene transfection for 3 days and the inhibition efficiency was obivious in 4th day. In the 5th day, the VEGF translation signal was hardly detected. Conclusion The antisense VEGF165 adenovirus gene transfection can inhibite not only the transcription but the translation of VEGF gene in synovial cells. Thus it can inhibit the secretion of VEGF in synovial cells with high efficiency.

Objective To evaluate the relationship between NT-probrain natriuretic peptide (NT-proBNP) levels and the degree of coronary artery stenosis in elderly patients with non-ST elevation acute coronary syndromes (NST-ACS).Methods The levels of NT-proBNP were determined in 258 elderly patients with NST-ACS divided into 3 quartile groups based on the degree of coronary artery stenosis,and 62 normal controls.And NT-pro BNP were compared among 4 groups.Results The serum levels of NT proBNP were increased in the NST-ACS patients with single,two or three-artery lesion compared with normal controls [(197.3±80.2)ng/L,(381.7±73.5)ng/L,(496.5± 99.8) ng/L vs.(68.2 ± 36.1) ng/L].The level of NT proBNP was enhanced along with increasing severity coronary artery disease (all P＜0.01).And with aging,the NT-proBNP levels were rising [aged＞60-69 years:(182.34±69.13) ng/L; aged≥70-79 years:(302.68±87.51)ng/L; aged≥80 years:(482.09±82.2)ng/L] (all P＜0.01).Conclusions The NT-proBNP level is enhanced along with aging and increasing severity of coronary artery stenosis in elderly NST-ACS patients.

Objective To investigate the expression of preferentially expressed antigen of melanoma (PRAME) gene in acute leukemia and its clinical significance.Methods PRAME mRNA levels were detected in bone marrow samples from 119 cases with acute leukemia (out of which 97 cases were acute myeloid leukemia, 22 cases were acute lymphocytic leukemia) by TaqMan based real-time quantitative PCR methods and compared it with the expression of FLT3 gene. Results PRAME mRNA was revealed in 50 of 97 AML (51.5 %), 8 of 22 ALL (36.4 %), and 5 patients were found little expression of PRAME in 11 healthy subjects and 17 non-blood disease patients. However, 17 AML, and 3 ALL patients with PRAME expression had no detectable FLT3. 9 patients with the expression of both PRAME and FLT3 genes were monitored in short follow-up. The lower level of PRAME was detected in all patients when they were in complete remission (CR) after chemotherapy. Conclusion PRAME gene was expressed in acute leukemia patients and will hopefully become a symbolic gene during the course of diagnosis and treatment in acute leukemia.

Objective To develop a recognition method of liver steatosis degree on type-B ultrasonic images based on multi-fractal spectrum texture analysis method and pattern recognition. Methods Features of singularity strength width and multi-spectrum area were extracted from the curve of multi-fractal spectrum of each liver ultrasonic images. These two features and the feature of mean intensity ratio comprised a three-dimensional feature vector, which would be classified by BP neural network. Results The classification accuracy was 96.00% for normal liver, 80.00% for mild fatty liver, 88.00% for moderate fatty liver and 92.00% for severe fatty liver. Conclusion Feature vector combined with BP neural network can identify the steatosis degree of liver on the ultrasonic images and can be used as an assistant diagnostic method.