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  1. Advanced Targeting Strategies for Murine Retroviral and Adeno-associated Viral Vectors

    Targeted gene delivery involves broadening viral tropism to infect previously nonpermissive cells, replacing viral tropism to infect a target cell...
    Julie H. Yu, David V. Schaffer in Gene Therapy and Gene Delivery Systems
    Chapter
  2. Nonviral Delivery of Cancer Genetic Vaccines

    The potential use of genetic vaccines to address numerous diseases including cancer is promising, but currently unrealized. Here, we review...
    Steven R. Little, Robert Langer in Gene Therapy and Gene Delivery Systems
    Chapter
  3. Production and Formulation of Adenovirus Vectors

    Adenovirus vectors have attracted considerable interest over the past decade, with ongoing clinical development programs for applications ranging...
    Nedim E. Altaras, John G. Aunins, ... Jayanthi J. Wolf in Gene Therapy and Gene Delivery Systems
    Chapter
  4. Molecular Conjugates

    Molecular conjugates are nanometer-sized entities consisting of synthetic materials (lipids, polycations, targeting agents, and so on) and nucleic...
    Jeremy Heidel, Swaroop Mishra, Mark E. Davis in Gene Therapy and Gene Delivery Systems
    Chapter
  5. Lentiviral Vectors

    We review the use of lentiviral vectors in current human gene therapy applications that involve genetic modification of nondividing tissues with...
    Nils Loewen, Eric M. Poeschla in Gene Therapy and Gene Delivery Systems
    Chapter
  6. Plasmid Vaccines and Therapeutics: From Design to Applications

    In the late 1980s, Vical and collaborators discovered that the injection into tissues of unformulated plasmid encoding various proteins resulted in...
    Marston Manthorpe, Peter Hobart, ... Alain Rolland in Gene Therapy and Gene Delivery Systems
    Chapter
  7. Adeno-associated Virus as a Gene Therapy Vector: Vector Development, Production and Clinical Applications

    Adeno-associated virus (AAV) has emerged as an attractive vector for gene therapy. AAV vectors have successfully been utilized to promote...
    Joshua C. Grieger, Richard J. Samulski in Gene Therapy and Gene Delivery Systems
    Chapter
  8. An integrated clinical approach to children at genetic risk for neurodevelopmental and psychiatric conditions: interdisciplinary collaboration and research infrastructure

    Background

    A sizeable proportion of pathogenic genetic variants identified in young children tested for congenital differences are associated with...

    Jane Summers, Danielle Baribeau, ... Jacob Vorstman in Journal of Neurodevelopmental Disorders
    Article Open access 05 July 2024
  9. Germline genome editing of human IVF embryos should not be subject to overly stringent restrictions

    This paper critiques the restrictive criteria for germline genome editing recently proposed by Chin, Nguma, and Ahmad in this journal. While praising...

    Article Open access 05 July 2024
  10. Is the Homologous Recombination Repair Mutation Defined by a 15-Gene Panel Associated with the Prognosis of Epithelial Ovarian Cancer?

    Background

    There is no consensus regarding the specific genes included in the homologous recombination repair (HRR) gene panel for identifying the HRR...

    Yi Liu, **aojun Chen, ... Ying Zhou in Molecular Diagnosis & Therapy
    Article 05 July 2024
  11. GSTO1 aggravates EGFR-TKIs resistance and tumor metastasis via deglutathionylation of NPM1 in lung adenocarcinoma

    Despite significantly improved clinical outcomes in EGFR-mutant lung adenocarcinoma, all patients develop acquired resistance and malignancy on the...

    Ning-xiang Shen, Ming-yu Luo, ... Ying Shen in Oncogene
    Article 05 July 2024
  12. The Relationship Between GAPDH Gene Polymorphism and Risk of Acute Coronary Syndrome in South Indians with Type 2 Diabetes Mellitus

    As glyceraldehyde-3-phosphate dehydrogenase (GAPDH) is one of the regulators of carbonyl stress, a pathogenic mechanism for diabetic complications...

    Sushmita Bora, Prashant Shankarrao Adole, ... Shaheer Ahmed in Biochemical Genetics
    Article 05 July 2024
  13. The Adaptive Role of Bark in the Diet of Budongo Chimpanzees (Pan troglodytes schweinfurthii)

    The ingestion of bark has been observed across the animal kingdom and is well documented in free-ranging chimpanzees. Thus far, the best-supported...

    E. Freymann, G. Badihi, ... S. Carvalho in International Journal of Primatology
    Article Open access 05 July 2024
  14. Genetic evidence for causal association between migraine and dementia: a mendelian randomization study

    Background

    There is an association between migraine and dementia, however, their causal relationship remains unclear. This study employed...

    Qiuyi Chen, Chengcheng Zhang, ... Lu Liu in BMC Medical Genomics
    Article Open access 05 July 2024
  15. Incorporating slow NMDA-type receptors with nonlinear voltage-dependent magnesium block in a next generation neural mass model: derivation and dynamics

    We derive a next generation neural mass model of a population of quadratic-integrate-and-fire neurons, with slow adaptation, and conductance-based...

    Hiba Sheheitli, Viktor Jirsa in Journal of Computational Neuroscience
    Article Open access 05 July 2024
  16. Validation of Nanopore long-read sequencing to resolve RPGR ORF15 genotypes in individuals with X-linked retinitis pigmentosa

    X-linked retinitis pigmentosa (XLRP) is characterized by progressive vision loss leading to legal blindness in males and a broad severity spectrum in...

    Christel Vaché, Valérie Faugère, ... Anne-Françoise Roux in European Journal of Human Genetics
    Article 05 July 2024
  17. Saturation genome editing of BAP1 functionally classifies somatic and germline variants

    Many variants that we inherit from our parents or acquire de novo or somatically are rare, limiting the precision with which we can associate them...

    Andrew J. Waters, Timothy Brendler-Spaeth, ... David J. Adams in Nature Genetics
    Article Open access 05 July 2024
  18. Saturation genome editing maps the functional spectrum of pathogenic VHL alleles

    To maximize the impact of precision medicine approaches, it is critical to identify genetic variants underlying disease and to accurately quantify...

    Megan Buckley, Chloé Terwagne, ... Gregory M. Findlay in Nature Genetics
    Article Open access 05 July 2024
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