keyword
MENU ▼
Read by QxMD icon Read
search

"Gene therapy"

keyword
https://www.readbyqxmd.com/read/28538131/gene-therapy-in-a-patient-with-sickle-cell-disease
#1
Philippe Leboulch, Marina Cavazzana
No abstract text is available yet for this article.
May 25, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28538130/gene-therapy-in-a-patient-with-sickle-cell-disease
#2
LETTER
(no author information available yet)
No abstract text is available yet for this article.
May 25, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28534887/when-is-early-antiretroviral-therapy-early-enough-for-hiv-remission
#3
Eva Poveda, Manuelo Cresp
Several strategies to reduce and control the HIV reservoir are being evaluating to achieve the great challenge of HIV remission or functional cure. Some of these are based on immune and gene therapy, or in the use of agents with latency reversing properties. Moreover, acute HIV infection and the impact of early antiretroviral treatment (ART) initiation is another key issue of current research. It has been demonstrated that ART initiation during acute HIV infection minimizes the establishment of the latent HIV reservoir...
May 23, 2017: AIDS Reviews
https://www.readbyqxmd.com/read/28534255/crispr-cas9-system-a-powerful-technology-for-in-vivo-and-ex-vivo-gene-therapy
#4
REVIEW
Xiaohui Zhang, Liren Wang, Mingyao Liu, Dali Li
CRISPR/Cas9 is a versatile genome-editing tool which is widely used for modifying the genome of both prokaryotic and eukaryotic organisms for basic research and applications. An increasing number of reports have demonstrated that CRISPR/Cas9-mediated genome editing is a powerful technology for gene therapy. Here, we review the recent advances in CRISPR/Cas9-mediated gene therapy in animal models via different strategies and discuss the challenges as well as future prospects.
April 20, 2017: Science China. Life Sciences
https://www.readbyqxmd.com/read/28533682/polyethylene-glycol-poly-%C3%AE%C2%B5-benzyloxycarbonyl-l-lysine-conjugated-vegf-sirna-for-antiangiogenic-gene-therapy-in-hepatocellular-carcinoma
#5
Gangmin Wang, XiaoLong Gao, GuoJun Gu, ZhiHong Shao, MingHua Li, PeiJun Wang, JianRong Yang, XiaoJun Cai, YongYong Li
A polyethylene glycol-poly(ε-benzyloxycarbonyl-l-lysine) (PEG-SS-PLL) block copolymer based on a disulfide-linked, novel biodegradable catiomer bearing a PEG-sheddable shell was developed to avoid "PEG dilemma" in nanoparticle intracellular tracking of PEG-PLL where PEG was nondegradable. However, PEG-SS-PLL catiomers have not been used to deliver small interfering VEGF RNA (siVEGF) in antiangiogenesis gene therapy. In this study, we aimed to investigate whether this novel biodegradable catiomer can deliver siVEGF into cancer cells and at the same time have an antitumor effect in a xenograft mouse model...
2017: International Journal of Nanomedicine
https://www.readbyqxmd.com/read/28532763/nanomaterial-enabled-cancer-therapy
#6
REVIEW
Sabina Quader, Kazunori Kataoka
While cancer remains the major cause of death worldwide, nanomaterial (NM)-based diagnosis and treatment modalities are showing remarkable potential to better tackle clinical oncology by effectively targeting therapeutic agents to tumors. NMs can selectively accumulate in solid tumors, and they can improve the bioavailability and reduce the toxicity of encapsulated cytotoxic agents. Additional noteworthy functions of NMs in cancer treatment include the delivery of contrast agents to image tumor sites, delivery of genetic materials for gene therapy, and co-delivery of multiple agents to achieve combination therapy or simultaneous diagnostic and therapeutic outcomes...
May 19, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28532005/non-viral-strategies-for-ocular-gene-delivery
#7
REVIEW
Ana V Oliveira, Ana M Rosa da Costa, Gabriela A Silva
The success of gene therapy relies on efficient gene transfer and stable transgene expression. The in vivo efficiency is determined by the delivery vector, route of administration, therapeutic gene, and target cells. While some requirements are common to several strategies, others depend on the target disease and transgene product. Consequently, it is unlikely that a single system is suitable for all applications. This review examines current gene therapy strategies, focusing on non-viral approaches and the use of natural polymers with the eye, and particularly the retina, as their gene delivery target...
August 1, 2017: Materials Science & Engineering. C, Materials for Biological Applications
https://www.readbyqxmd.com/read/28531117/immunobiology-of-newcastle-disease-virus-and-its-use-for-prophylactic-vaccination-in-poultry-and-as-adjuvant-for-therapeutic-vaccination-in-cancer-patients
#8
REVIEW
Volker Schirrmacher
Newcastle disease (ND) is one of the most important diseases of poultry worldwide. In the last decades, molecular research has gained a lot of new information about its causative agent, newcastledisease virus (NDV). In poultry industry, certain strains of NDV have been used for preventive vaccination for more than 60 years. NDV has also been applied to cancer patients with beneficial effects for about 50 years, but this is less well known. The molecular basis for these differential effects of NDV in birds and man have been elucidated in the last decades and are explained in this review...
May 20, 2017: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28530842/the-past-present-and-future-of-gene-therapy-from-nobel-laureate-david-baltimore
#9
James M Wilson
No abstract text is available yet for this article.
May 22, 2017: Human Gene Therapy. Clinical Development
https://www.readbyqxmd.com/read/28530652/cd34-cells-from-dental-pulp-stem-cells-with-a-zfn-mediated-and-homology-driven-repair-mediated-locus-specific-knock-in-of-an-artificial-%C3%AE-globin-gene
#10
S Chattong, O Ruangwattanasuk, W Yindeedej, A Setpakdee, K Manotham
In humans, mutations in the β-globin gene (HBB) have two important clinical manifestations: β-thalassemia and sickle cell disease. The progress in genome editing and stem cell research may be relevant to the treatment of β-globin-related diseases. In this work, we employed zinc finger nuclease (ZFN)-mediated gene integration of synthetic β-globin cDNA into HBB loci, thus correcting almost all β-globin mutations. The integration was achieved in both HEK 293 cells and isolated dental pulp stem cell (DPSCs)...
May 22, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28530226/corrigendum-cochlear-gene-therapy-with-ancestral-aav-in-adult-mice-complete-transduction-of-inner-hair-cells-without-cochlear-dysfunction
#11
Jun Suzuki, Ken Hashimoto, Ru Xiao, Luk H Vandenberghe, M Charles Liberman
This corrects the article DOI: 10.1038/srep45524.
May 22, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28530135/prolonged-expression-of-secreted-enzymes-in-dogs-after-liver-directed-delivery-of-i-sleeping-beauty-i-transposons-implications-for-non-viral-gene-therapy-of-systemic-disease
#12
Elena L Aronovich, Kendra Anne Hyland, Bryan C Hall, Jason B Bell, Erik R Olson, Myra Urness Rusten, David W Hunter, N Matthew Ellinwood, R Scott McIvor, Perry B Hackett
The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice including hemophilias A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis (MPS) types I and VII caused by α-L-iduronidase (IDUA) and β-glucuronidase (GUSB) deficiency, respectively. We recently reported modified approaches of the hydrodynamics-based procedure to deliver transposons to the liver in dogs. Using the transgenic canine reporter secreted alkaline phosphatase (cSEAP), we demonstrated transgenic protein in the plasma for up to six weeks post-infusion...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28530128/in-vivo-endocrine-secretion-of-prostacyclin-following-expression-of-a-cox-1-pgis-fusion-protein-in-the-salivary-glands-of-rats-via-non-viral-gene-therapy
#13
Zhimin Wang, Raymond L Benza, Lee Zourelias, Angela Sanguino, Ramaz Geguchadze, Kelly J Shields, Changgong Wu, Kristin B Highland, Michael J Passineau
Pulmonary arterial hypertension is a progressive disease that culminates in right heart failure and death. Prostacyclin(PGI2) and its derivatives are effective treatments for PAH when administered as continuous parenteral infusions. This treatment paradigm requires medical sophistication, and patients are at risk for complications from an indewelling catheter; drug interruptions may result in rebound pulmonary hypertension and death. We hypothesized that the salivary gland can be repurposed into an endogenous production site for circulating PGI2 through the expression of a fusion protein embodying cyclooxygenase-1(Cox1) and prostacyclin synthase(PGIS) domains...
May 20, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28529903/immunotherapy-and-radiation-therapy-for-malignant-pleural-mesothelioma
#14
REVIEW
Evan W Alley, Sharyn I Katz, Keith A Cengel, Charles B Simone
Malignant pleural mesothelioma (MPM) is a particularly aggressive thoracic malignancy with limited survival following combination chemotherapy. As a result, there has been increased interested in immunotherapy for mesothelioma, both in the first-line and salvage settings. Early investigations of interleukin-2 (IL-2) and interferon alfa-2a/b have been limited by modest response rates and toxicity, whereas cytokine gene therapy is currently being investigated and shows early promise. The most prominent class of immunotherapies to be trialed with mesothelioma in the past half-decade has been immune checkpoint inhibitors (CPI)...
April 2017: Translational Lung Cancer Research
https://www.readbyqxmd.com/read/28529641/multifunctional-nucleus-targeting-nanoparticles-with-ultra-high-gene-transfection-efficiency-for-in-vivo-gene-therapy
#15
Ling Li, Xia Li, Yuzhe Wu, Linjiang Song, Xi Yang, Tao He, Ning Wang, Suleixin Yang, Yan Zeng, Qinjie Wu, Zhiyong Qian, Yuquan Wei, Changyang Gong
Cancer stem cell-like cells (CSCL) are responsible for tumor recurrence associated with conventional therapy (e.g. surgery, radiation, and chemotherapy). Here, we developed a novel multifunctional nucleus-targeting nanoparticle-based gene delivery system which is capable of targeting and eradicating CSCL. These nanoparticles can facilitate efficient endosomal escape and spontaneously penetrate into nucleus without additional nuclear localization signal. They also induced extremely high gene transfection efficiency (>95%) even in culture medium containing 30% serum, which significantly surpassed that of some commercial transfection reagents, such as Lipofectamine 2000 and Lipofectamine 3000 etc...
2017: Theranostics
https://www.readbyqxmd.com/read/28529622/ires-mediated-protein-translation-overcomes-suppression-by-the-p14arf-tumor-suppressor-protein
#16
Song Xi, Ming Zhao, Si Wang, Ling Ma, Shensen Wang, Xianling Cong, Ruth A Gjerset, Rebecca C Fitzgerald, Yinghui Huang
Internal ribosome entry sites (IRES elements) have attracted interest in cancer gene therapy because they can be used in the design of gene transfer vectors that provide bicistronic co-expression of two transgene products under the control of a single promoter. Unlike cellular translation of most mRNAs, a process that requires a post-translational 5' modification of the mRNA known as the cap structure, IRES-mediated translation is independent of the cap structure. The cellular conditions that may intervene to modulate IRES-mediated, cap-independent versus cap-dependent translation, however, remain poorly understood, although they could be critical to the choice of gene transfer vectors...
2017: Journal of Cancer
https://www.readbyqxmd.com/read/28529588/downregulation-of-calbindin-1-a-calcium-binding-protein-reduces-the-proliferation-of-osteosarcoma-cells
#17
Zhengxiang Huang, Guojun Fan, Dongliang Wang
Osteosarcoma is the most common type of primary malignant bone tumor and has a high propensity to metastasize to the lungs and bones. Calbindin 1 (CALB1) is a constituent Ca2+ binding protein, which can prevent apoptotic death in several cell types induced through various pro-apoptotic signaling pathways. To investigate whether CALB1 is implicated in the tumor growth of human osteosarcoma, two different short hairpin RNAs (shRNAs) against CALB1 were used for CALB1-knockdown in osteosarcoma U2OS cells. The U2OS cells were divided into three groups: Two groups with CALB1 knockdown (CALB1-shRNA 1 and CALB1-shRNA 2) and one control group (Con-shRNA)...
May 2017: Oncology Letters
https://www.readbyqxmd.com/read/28529585/efficacy-of-recombinant-human-adenovirus-p53-combined-with-chemotherapy-for-locally-advanced-cervical-cancer-a-clinical-trial
#18
Jie Xiao, Jian Zhou, Min Fu, Li Liang, Qiaoling Deng, Xiaoyun Liu, Fumin Liu
The aim of this study was to evaluate the efficacy and safety of recombinant human adenovirus-p53 (rhAd-p53) combined with neoadjuvant chemotherapy in treatment of locally advanced cervical cancer (LACC). A total of 40 patients with LACC (stage IB2 to IIIA) were randomized into 2 groups (n=20 each): PVB group (cisplatin + vincristine + bleomycin, intravenously) and combined group (rhAd-p53 gene therapy + neoadjuvant chemotherapy). Both groups underwent a course of chemotherapy; the only exception was the injection of the rhAd-p53 solution 1×10(12) VP intratumorally at an interval of three days thrice in the combined group thereafter...
May 2017: Oncology Letters
https://www.readbyqxmd.com/read/28529584/anti-microrna-21-221-and-microrna-199a-transfected-by-ultrasound-microbubbles-induces-the-apoptosis-of-human-hepatoma-hepg2-cells
#19
Xinmin Guo, Shunhua Guo, Liwen Pan, Li Ruan, Yingshi Gu, Jichuang Lai
Gene therapy, particularly microRNA (miRNA), is a promising candidate in the treatment of cancer; however, it is challenging to develop gene delivery systems. Ultrasound microbubbles have been used for gene delivery with excellent results. The present study aimed to investigate the transfection efficiency of HepG2 cells using ultrasound microbubbles. The effects of three miRNAs (miR-21, miR-221 and miR-199a) on HepG2 cells were also determined by performing ultrasound microbubble-mediated gene transfection...
May 2017: Oncology Letters
https://www.readbyqxmd.com/read/28529412/accelerated-wound-healing-harnessing-the-power-of-platelets-biomaterials-stem-cells-and-gene-therapy
#20
EDITORIAL
Niti Khunger
No abstract text is available yet for this article.
January 2017: Journal of Cutaneous and Aesthetic Surgery
keyword
keyword
29036
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"