The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene therapy advances.
Gene therapy is a new form of molecular medicine that holds tremendous promise for the treatment of human infectious diseases. Perhaps no better example of the broad impact of gene therapy for the treatment of human infectious diseases comes from studies on the human immunodeficiency virus, HIV 1...
19.8Impact Factor Structure Supportsopen access Trends in Endocrinology & Metabolism Supportsopen access Trends in Biochemical Sciences Supportsopen access Trends in Biotechnology Supportsopen access Trends in Cell Biology Supportsopen access Trends in Cognitive Sciences ...
doses, likely to achieve clinical efficacy, thereby increasing the safety as well as reducing vector production costs, ensuring translation to the clinic with higher probability of success, without the need for the use of immune suppression, for gene therapy of a wide variety of diseases in ...
K. Human population-specific gene expression and transcriptional network modification with polymorphic transposable elements. Nucleic Acids Res. 45, 2318–2328 (2017). CAS PubMed Google Scholar Cordaux, R. & Batzer, M. A. The impact of retrotransposons on human genome evolution. Nat. Rev. ...
(modelling atezolizumab + bevacizumab as first-line HCC systemic therapy10) did not impact the overall survival in this cohort (Fig.4gand Extended Data Fig.9a,b). These results are similar to the reported drug responses to TKIs and ICI in human patients with activated β-catenin signalling5....
I find it difficult to describe in words - the deeds speak for themselves - the impact with which clinical cytogene- tics inflamed and revolutionized and ultimately changed the very framework and fabric of human genetics. But let a human geneticist speak. In 1960, commenting on the Denver ...
et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet.2000;24:257-261.Google Scholar 24. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G. et al. Gene therapy of human severe combined immunodeficiency (SCID)-...
Phenylalanine ammonia lyase (PAL) is able to convert phenylalanine into ammonia and trans-cinnamic acid, which serves as a potential enzyme for PKU therapy [41]. A large amount of PAL protein was obtained by heterologous over-expression of PAL gene from Rhodosporidium toruloides in Escherichia ...
New Drug Repositioning (vt.使复位;改变…的位置;使变换阵地,n.复位;贮藏;放回)Candidates for T-ALL Identified Via Human/Murine Gene Signature Comparison T-cell Acute Lymphoblastic Leukemia (T-ALL) is an aggressive subtype of leukemia for which important progress in treatment efficiency have been mad...