Global ETD Search
Search theses and dissertations gathered from participating repositories worldwide. Every result links back to the library that holds it. No account is needed.
Results
Showing 1 to 20 of 383 for “"gene therapy"”.
-
Applications of mitochondrial gene therapy
Genetic aberrations in the mitochondrial genome (mtDNA) can often manifest as clinical pathologies. Along with genetic mutations in nuclear encoded mitochondrial genes, these pathologies form a group of genetic disorders referred to as mitochondrial diseases. Engineered mitochondrially targeted …
-
Gene therapy for axon regeneration
… in the central nervous system (CNS) do not regenerate. Traumatic CNS injuries are a significant clinical problem and have life-changing consequences for patients. There are 130.000 new cases of spinal cord injury (SCI) per year worldwide and currently affects more than 2.5 million people …
-
Multifunctional bioreducible nanoparticles for gene therapy
<p>Gene therapy is a promising therapeutic strategy to treat diseases caused by single or multiple gene mutations. Non-viral gene delivery vectors exhibit many advantages over viral vectors, including enhanced safety, versatility in choosing different types of therapeutic nucleic acids, and ease of …
-
Novel Approaches to Thalassaemia Gene Therapy
The haemoglobinopathies are the most prevalent genetically inherited disorders worldwide. Gene therapy for p-thalassaemia is particularly challenging given the requirement for high p-globin chain production in a lineage-specific manner. Advances in lentiviral vector (LV) technology, together with …
-
Leveraging Genomic Information for Safer Gene Therapy
<p>Gene therapy holds enormous potential for the treatment of genetic disorders, which are caused by mutations in DNA disrupting normal gene function. These mutations can be monogenic, affecting a single gene, or polygenic, involving multiple genes. Since, the first gene therapy treatment for SCID …
-
Engineering of Nonviral Vectors for Gene Therapy
Finally, for polymeric devices to be made safe and efficient for in vivo usage there must be some means to target them to specific cells or tissues. Various cyclodextrins were conjugated to PEI using an amide linkage. The hydrophobic interior of the cyclodextrin allows the facile addition of small …
-
Magnetic targeted drug delivery system in gene therapy
… for a wide range of medical applications. Gene therapy is the insertion of genes into an individual's cells and tissues to treat diseases. As the recombinant virus vector has many limits and problems, more studies turn to the nonvirus vectors, which have transfection efficiency and low …
-
Targeted gene therapy for canine osteosarcoma: preliminary investigations
… and <20% survive >2 years with standard therapy. Hence, new approaches must be considered. TP53 is altered in ~50% of human and canine cancers, including OS, making it a candidate for targeted suicide gene therapy strategies. Canine OS is considered to be a good model for human OS. The …
-
Preclinical Assessment of Intravenous Gene Therapy for GM2 Gangliosidosis
GM2 Gangliosidoses are a group of severe neurodegenerative lysosomal storage disorders characterized by the inability to catabolize GM2 Gangliosides, leading to a neurotoxic accumulation of the GM2 lipids within the central nervous system. The Hexosaminidase A (HexA) enzyme is a heterodimeric …
-
Development of A Gene Therapy for Creatine Transporter Deficiency
… creatine deficiency syndromes are a group of genetic disorders caused by the inability to synthesize or transport creatine, leading to a severe depletion of creatine in the brain. Creatine transporter deficiency is one of the three Cerebral creatine deficiency syndromes caused by loss of …
-
Improving Viral Vectors for Gene Targeting in Gene Therapy
… one out of every hundred live births (WHO). Gene targeting is a term that is used describe the manipulation of genetic material, either by adding a gene in a specific locus, creating a mutation at a specific locus, or correcting a gene at a specific locus. Here, unless otherwise noted, we …
-
Proteasome Inhibition Enhances The Effect of Bikdd Gene Therapy
… proteasome inhibition as a combinational therapy strategy with BikDD gene therapy to enhance the clinical benefits. Proteasome inhibition stabilized exogenously expressed BikDD protein, and significantly enhanced the apoptosis induction effect of BikDD. Studies on detailed mechanism for …
-
Novel non-viral delivery systems for cancer gene therapy
The non-viral gene delivery systems dendrimers have been widely studied for cancer therapy. However, due to lack of targeting efficacy, their therapeutic applications are still limited. In this present study, our aim was to investigate the efficacy of four formulations of modified generation- 3- …
-
Gene therapy in mouse models of spinal muscular atrophy :
… recessive neuromuscular disorder, is the leading genetic cause of infant mortality. SMA is caused by the functional, homozygous loss of the Survival Motor Neuron-1 (SMN1) gene which encodes for the ubiquitously expressed Survival Motor Neuron (SMN) protein. Here we utilize SMA mouse models to …
-
Gene therapy for epilepsy: resculpturing synaptic transmission with neuropeptides
… for viral vector-mediated transfer of gene and expression of NPY in epileptogenic regions of the brain, providing effective suppression of both acute and chronic seizures in animal models of epilepsy. Therefore, NPY gene therapy strategies are currently under intense investigation and …
-
Ionizing Radiation and Endostatin Gene Therapy Against Lung Tumor
<p>Although many advances have been made in radiotherapy, tumor radioresistance and normal tissue damage continue to always be important issues for radiation oncologists. Numerous reports have also documented that radiation exposure increases the risk for malignancy and suppresses immune …
-
Combined Cell and Gene Therapy Towards the Prevention of Osteoarthritis
… and no approved medications, biological therapy, or procedure cures or prevents the progressive destruction of the joint. Present treatment options primarily offer symptomatic relief rather than preventative or regenerative results. There is an urgent call to innovate, validate, and …
-
IL -10 gene therapy for the treatment of pulmonary inflammation
… the potential therapeutic effect of IL-10 gene therapy for the treatment of pulmonary inflammation and to develop appropriate gene delivery and expression systems for efficient gene transfer to the lung. We evaluated various liposomal and non-liposomal agents including LipofectAMINE RTM, …
-
Gene therapy in a new model of delayed wound healing
… healing. Furthermore we hypothesized that cell therapy and gene therapy may enhance epidermal healing in this model of delayed wound healing. In study one, we establish a delayed wound healing model in yorkshire pigs, mimicking diabetic wound healing, by injecting streptozotocin intravenously. …
-
Targeting gene therapy to neuroinfalmmatory lesions in experimental autoimmune encephalomyelitis
… (Lin-BM) vom Knochenmark β-Aktin-GFP transgener Mäuse isoliert. Eine Transplantation dieser markierten Stammzellen (GFP+Lin-BM) in Mäuse, die vorher einer letalen Strahlendosis ausgesetzt waren, führte zu deren Aufnahme in nicht-hematopoetisches Gewebe einschließlich des Gehirns und des …
Page 1 of 20