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.
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Showing 1 to 20 of 41 for “"Gene therapies"”.
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Development of gene therapies for treatment of neurological disorders
Submission published under a 24 month embargo labeled 'Closed Access', the embargo will last until 2027-05-01
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Engineered synthetic translational control for next generation mRNA gene therapies
… mRNA is an emerging therapeutic modality for gene and cell therapy. Unlike their synthetic DNA counterparts, synthetic mRNA has an increased safety profile due to its transient gene expression and ability to express outside of the nucleus. Furthermore, it can be more easily delivered to cells …
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Tumor Response TCF-4/β-Catenin Regulatory Elements for Enhancing Cancer Gene Therapies
<p>Mutations in the adenomatous polyposis coli gene are frequently associated with progression of colon carcinoma and most other types of epithelial carcinomas. This usually results in stabilization of β-catenin protein levels, followed by transactivation of Tcf-4/β-catenin responsive genes. The …
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ADVANCED AAV-MEDIATED LIVER-DIRECTED GENE THERAPIES FOR HAEMOPHILIA A AND MUCOPOLYSACCHARIDOSIS TYPE VI
Liver-directed gene therapy using adeno-associated viral (AAV) vectors holds significant promise for providing long-term transgene expression following a single systhemic administration. However, both the limited cargo capacity (~4.7 kb) and the non-integrative nature of AAV vectors prevent their …
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Leveraging digital tools and analytics for temperature management in cold chain systems for gene therapies
Emerging advanced therapies at Johnson & Johnson Innovative Medicine, such as a new retina gene therapy, require maintaining ultra low temperatures within the cold supply chain from the manufacturing plant and throughout distribution to the customer. In comparison to traditional cold chain …
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Future Preventive Gene Therapy of Polygenic Diseases from a Population Genetics Perspective
… the accumulation of scientific knowledge of the genetic causes of common diseases and the continuous advancement of gene-editing technologies, gene therapies to prevent polygenic diseases may soon become possible. This research assesses the population genetic consequences of such therapies. This …
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The potential of exosome-based gene therapy to eradicate glioblastoma cells
… have emerged as a new potential carrier for gene therapies in cancer treatment due to their natural material transport properties, biocompatibility, and specificity in transporting cargo to the target cells. These extracellular vesicles have the additional advantage of being able to cross the …
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Stochastic modeling of performance-based annuities: increasing gene therapy accessibility by managing the uncertainty of costs and treatment value
Durable gene therapies are a new and upcoming form of treatment. They have a high prices and high treatment uncertainty. Payers may feel reluctant to pay for such treatments with traditional financing methods because they are concerned about treatment performance risk, actuarial risk, and payment …
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An Economic and Regulatory Analysis of Gene Therapy Products Authorized by the US Food and Drug Administration and the European Medicines Agency
<p>Background: Gene therapy has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies.</p> <p>Objective: This study assessed the authorization process of gene therapy products by the Food and …
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Engineering minimally immunogenic cargos and delivery modalities for gene therapy
Since the discovery of CRISPR-Cas9 systems, gene therapies have revolutionized the field of molecular biology by introducing functional genes into cells to correct genetic defects or diseases. To date, several gene therapies are pending approval for use in the clinic and have shown promise in the …
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Looking at the Map, Together: Modeling Treatment Center Location Selection and its Effects on Access to Gene Therapy in Brazil
… how many and which treatment centers to offer a gene therapy to patients is a crucial decision which impacts how far the treatment has to be transported and how far patients have to travel to receive treatment. Many gene therapies are for patients with severe diseases that make it difficult to …
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Development of high-throughput nanoparticle screening technologies to facilitate the delivery of genetic therapies to non-liver cell types
Genetic drugs (such as siRNAs, mRNAs, and CRISPR/Cas9) have the potential to be curative therapies for countless diseases. However, gene therapies will only work if the genetic drug is delivered to the diseased cell type. One promising delivery method is through the use of Lipid Nanoparticles …
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Cross-Species Evolution of New AAV Variants
<p>Therapeutic gene transfer and genome editing require effective delivery of genetic cargo to target cells and tissues. Recombinant adeno-associated viral (AAV) vectors are a promising delivery platform, but ongoing clinical trials continue to highlight a relatively narrow therapeutic window. …
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Overcoming Challenges in Cellular Therapies: A Systems Engineering Approach for Equitable Access
Cellular and gene therapies have ushered in a new era of medical treatment, promising cures previously thought unattainable. Technologies like CRISPR/Cas9 enable precise genome manipulation, yet challenges persist in therapy delivery, prompting the rise of ex vivo approaches. Despite the promise of …
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Design of Human Serum Albumin and Adenovirus Conjugation via Catcher/Tag Molecular Glue
… response makes it ideal for in vivo gene therapies since its discovery in 1953. However, the robust immunogenicity of the Ad capsid and low vaccine absorption via mucous membranes and epithelium put a limit on the process of developing intranasal vaccines. Efforts are being made to …
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Development of Extrachromosomal Genetic Technologies for Persistent and Tunable Expression of Therapeutic Payloads
Effective cell and gene therapies (CGT) require sustained expression of therapeutic payloads, typically achieved through genomic integration of transgenic DNA via genome editing (e.g., CRISPR\Cas9) or integrating viral vectors (e.g., retrovirus, lentivirus). Integration-based approaches risk …
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Hematopoietic Stem Cell Threshold Sensing Controls Regulatory Pathways Facilitating Clinically Relevant Ex Vivo Expansion for Stem Cell Transplantation
… administering a larger pool of HSCs. 3.) Lastly, gene therapies for hematological diseases still require a robust supply of HSCs to offset varying degrees of inefficiency in vector mediated transfection protocols. These reasons, and others, have been an impetus for many discoveries made within …
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REMOTE CONTROL OF CELL FUNCTION USING TEMPERATURE AS AN INPUT
The increasing prevalence of RNA and cell-based therapies such as CAR-T, encapsulated cells, and gene therapies have generated a need to control cell function deep inside the body. However, traditional methods of deep-tissue cell actuation require injection of chemicals which can suffer from poor …
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Analysis of the role of lipids in retrovirus transduction
The most common gene transfer vehicle used in gene therapy protocols are mammalian virus vectors. Specifically, retroviruses are one of the most common viral vectors used since they are able to permanently integrate their transgene into the host cell genome, providing, in principal, to a long-term …
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Engineering Viruses for Gene Therapy: Isolating and Characterizing Murine Leukemia Virus With Improved Stability
Inefficient gene delivery continues to be a primary hurdle facing gene therapy. Viruses offer the highest gene transfer capabilities but are not optimized as therapeutics. Applying directed evolution, we randomly mutated the entire genome of amphotropic murine leukemia virus (MLV) and selected for …
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