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Latest Articles on Gene Therapy

Overview of latest articles and publications on gene therapy in PubMed, including Human Gene Therapy, Journal of Molecular Medicine and Journal of Gene Medicine. PubMed is a service of the US National Library of Medicine that includes over 18 million citations from MEDLINE and other life science journals.


  • Muscle activations during gait in children with Duchenne muscular dystrophy.
    Muscle activations during gait in children with Duchenne muscular dystrophy. [JOURNAL ARTICLE]Ann Phys Rehabil Med 2016 Sep.:e82-e83.APRopars J, Lempereur M, Brochard S, et al. Since motor commands are normal in DMD, the hyper-activity and co-contractions are likely to compensate for gait instability and muscle weakness, however, they may have negative consequences on the mus...Publisher Full TextThe aim of this prospective study was to investigate changes in muscle activity during gait in children with Duchenne muscular Dystrophy (DMD).Dynamic surface electromyography recordings (EMGs) of 16 children with DMD and pathological gait were compared with those of 15 control children. The activity of the rectus femoris (RF), vastus lateralis (VL), medial hamstrings (HS), tibialis anterior (TA) and gastrocnemius soleus (GAS) muscles was recorded and analyzed quantitatively and qualitatively.Overall muscle activity in the children with DMD deviated significantly from that of the control group. Percentage activation amplitude of RF, HS and TA was greater throughout the gait cycle in the children with DMD while the timing of GAS activity differed from the control children. Significantly, greater muscle coactivation was found in the children with DMD. There were no significant differences between sides.Since motor commands are normal in DMD, the hyper-activity and co-contractions are likely to compensate for gait instability and muscle weakness, however, they may have negative consequences on the muscles and may increase the energy cost of gait. Simple rehabilitative strategies such as targeted physical therapy or the use of a light ankle foot orthosis may improve stability and thus the pattern of muscle activity.

  • Modified mRNA for BMP 2 in combination with biomaterials serves as a transcript activated matrix for effectively inducing osteogenic pathways in stem cells.
    Modified mRNA for BMP 2 in combination with biomaterials serves as a transcript activated matrix for effectively inducing osteogenic pathways in stem cells. [JOURNAL ARTICLE]Stem Cells Dev 2016 Sep 27.SCBalmayor ER, Geiger JP, Koch C, et al. Bone regeneration using stem cells and growth factors has disadvantages while needing to use supraphysiological growth factors concentrations. Gene therapy has been proposed as alternative, but also ha...Publisher Full TextPublisher Full TextBone regeneration using stem cells and growth factors has disadvantages while needing to use supraphysiological growth factors concentrations. Gene therapy has been proposed as alternative, but also has limitation. mRNA based transcript therapy is a novel approach that may solve pDNA based gene therapy limitations. Although much more efficient in delivering genes into the cell, mRNA is unfortunately unstable and immunogenic. However, recent reports indicated that chemical modifications of the mRNA molecule can improve stability and toxicity. In this study, we have combined biomaterials and chemically modified mRNA (cmRNA) encoding Metridia luciferase, eGFP, and BMP 2 in order to develop transcript activated matrices (TAMs) for gene transfer to stem cells. BMP 2 cmRNA was produced to evaluate its feasibility in stimulating osteogenic differentiation. Fibrin gel and calcium phosphate granules (MBCP) were used as biomaterials. A sustained release of hBMP 2 cmRNA from both biomaterials was observed during 7 days. This occurred significantly faster from the MBCP granules compared to fibrin gels (92% from MBCP and 43% from fibrin after 7 days). Stem cells cultured in hBMP 2 cmRNA/fibrin or on hBMP 2 cmRNA/MBCP were transfected and able to secrete significant amounts of hBMP 2. Furthermore, transfected cells expressed osteogenic markers in vitro. Interestingly, although both TAMs promoted gene expression at the same level, hBMP 2 cmRNA/MBCP granules induced significantly higher collagen I and osteocalcin gene expression. This matrix also induced more mineral deposition. Overall, our results demonstrated the feasibility of developing efficient TAMs for bone regeneration by combining biomaterials and cmRNAs. MBCP synergistically enhances the hBMP 2 cmRNA induced osteogenic pathway.

  • Pharmacogenomic approaches to lipid-regulating trials.
    Pharmacogenomic approaches to lipid-regulating trials. [JOURNAL ARTICLE]Curr Opin Lipidol 2016 Sep 26.COBertrand MJ, Dubé MP, Tardif JC Pharmacogenomics hold great potential in future lipid trials to decrease failure rates in drug development and to identify patients who will respond with greater benefits and smaller risk.Publisher Full TextRandomized clinical outcome trials are costly, long, and often yield neutral or modestly positive results, and these issues have impeded cardiovascular drug development in the past decade. Despite the significant reduction of cardiovascular morbidity and mortality with statins, substantial residual risk of major cardiovascular events remains. This could be because of the difficulty of demonstrating benefits of new drugs in addition to the current standard of care in unselected populations as well as the interindividual variability in drug response. Pharmacogenomics is a promising avenue for the development of novel or failed drugs and for the repurposing of other medications.Several variants were identified in genes that were associated with the effects of statins on plasma lipids. Genomic studies of mutations in genes that encode drug targets have the potential to inform on the link between drug therapy acting on those targets and clinical outcomes. Recently, ADCY9 gene variants were shown to be significantly associated with responses to dalcetrapib in terms of clinical outcomes, atherosclerosis imaging, cholesterol efflux, and inflammation, which provided support for the conduct of a new prospective clinical trial in a genetically determined population.Pharmacogenomics hold great potential in future lipid trials to decrease failure rates in drug development and to identify patients who will respond with greater benefits and smaller risk.

  • Identifying High-Risk N0 and N1 Breast Cancer Patients for Postmastectomy Radiation Therapy by an 18-Gene Classifier.
    Identifying High-Risk N0 and N1 Breast Cancer Patients for Postmastectomy Radiation Therapy by an 18-Gene Classifier. [JOURNAL ARTICLE]Int J Radiat Oncol Biol Phys 2016 Oct 1; 96(2S):S146-S147.IJCheng SH Publisher Full Text

  • The 21-Gene Recurrence Score and Locoregional Recurrence Rates in Patients With Node-Positive Breast Cancer Treated on SWOG S8814.
    The 21-Gene Recurrence Score and Locoregional Recurrence Rates in Patients With Node-Positive Breast Cancer Treated on SWOG S8814. [JOURNAL ARTICLE]Int J Radiat Oncol Biol Phys 2016 Oct 1; 96(2S):S146.IJWoodward WA, Barlow WE, Jagsi R, et al. Publisher Full Text

  • A 24-Gene Predictor of Response to Postoperative Radiation Therapy in Prostate Cancer.
    A 24-Gene Predictor of Response to Postoperative Radiation Therapy in Prostate Cancer. [JOURNAL ARTICLE]Int J Radiat Oncol Biol Phys 2016 Oct 1; 96(2S):S104-S105.IJZhao SG, Chang SL, Spratt DE, et al. Publisher Full Text

  • Using Genetic Algorithms in Mathematical Modeling of Optimal Radiation Therapy Schedules for GBM.
    Using Genetic Algorithms in Mathematical Modeling of Optimal Radiation Therapy Schedules for GBM. [JOURNAL ARTICLE]Int J Radiat Oncol Biol Phys 2016 Oct 1; 96(2S):E73.IJHathout L, Shamma J Publisher Full Text

  • A Prognostic Model Combining Genetic Variations in the Transforming Growth Factor-Beta1 Pathway and Clinical Factors for Non-Small Cell Lung Cancer After Radiation Therapy.
    A Prognostic Model Combining Genetic Variations in the Transforming Growth Factor-Beta1 Pathway and Clinical Factors for Non-Small Cell Lung Cancer After Radiation Therapy. [JOURNAL ARTICLE]Int J Radiat Oncol Biol Phys 2016 Oct 1; 96(2S):E667.IJZhang H, Jin JY, Wang W, et al. Publisher Full Text

  • Genetic and Non-Genetic Factors Affecting The Response To Clopidogrel Therapy.
    Genetic and Non-Genetic Factors Affecting The Response To Clopidogrel Therapy. [JOURNAL ARTICLE]Clin Ther 2016 Oct 6; 38(10S):e11.CTMugosa S, Sahman-Zaimovic M, Todorovic Z, et al. Publisher Full Text

  • SUPRASPINAL CONTROL PREDICTS LOCOMOTOR FUNCTION AND FORECASTS RESPONSIVENESS TO TRAINING AFTER SPINAL CORD INJURY.
    SUPRASPINAL CONTROL PREDICTS LOCOMOTOR FUNCTION AND FORECASTS RESPONSIVENESS TO TRAINING AFTER SPINAL CORD INJURY. [JOURNAL ARTICLE]J Neurotrauma 2016 Sep 27.JNField-Fote E, Yang JF, Basso DM, et al. Restoration of walking ability is an area of great interest in the rehabilitation of persons with spinal cord injury. Since many cortical, subcortical, and spinal neural centers contribute to locomotor...Publisher Full TextPublisher Full TextRestoration of walking ability is an area of great interest in the rehabilitation of persons with spinal cord injury. Since many cortical, subcortical, and spinal neural centers contribute to locomotor function, it is important that intervention strategies be designed to target neural elements at all levels of the neuraxis that are important for walking ability. While to date most strategies have focused on activation of spinal circuits, more recent studies are investigating the value of engaging supraspinal circuits. Despite the apparent potential of pharmacological, biological, and genetic approaches, as yet none have proved more effective than physical therapeutic rehabilitation strategies. By making optimal use of the potential of the nervous system to respond to training, strategies can be developed that meet the unique needs of each individual. To complement the development of optimal training interventions, it is valuable to have the ability to predict future walking function based on early clinical presentation, and to forecast responsiveness to training. A number of clinical prediction rules and association models based on common clinical measures have been developed with the intent, respectively, to predict future walking function based on early clinical presentation, and to delineate characteristics associated with responsiveness to training. Furthermore, a number of variables that are correlated with walking function have been identified. Not surprisingly, most of these prediction rules, association models, and correlated variables incorporate measures of volitional lower extremity strength, illustrating the important influence of supraspinal centers in the production of walking behavior in humans.

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