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  1. 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
  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. 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
  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. 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
  6. 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
  7. 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
  8. Beyond black-box models: explainable AI for embryo ploidy prediction and patient-centric consultation

    Purpose

    To determine if an explainable artificial intelligence (XAI) model enhances the accuracy and transparency of predicting embryo ploidy status...

    Thi-My-Trang Luong, Nguyen-Tuong Ho, ... Chii-Ruey Tzeng in Journal of Assisted Reproduction and Genetics
    Article 04 July 2024
  9. Monitoring of controlled ovarian stimulation in IVF

    Since the inception of in vitro fertilization (IVF), monitoring of controlled ovarian stimulation (COS) has traditionally involved numerous...

    Shahar Kol, Juan Carlos Castillo Farfan, ... Alexander M. Quaas in Journal of Assisted Reproduction and Genetics
    Article Open access 04 July 2024
  10. Chorio-retinal vessel density in women affected by functional hypothalamic amenorrhea: a monocentric observational cross-sectional study to evaluate the impact of hypoestrogenism on chorio-retinal vascularization

    Purpose

    Functional hypothalamic amenorrhea (FHA) is characterized by an estrogen deficiency which in turn can cause vascular dysfunction. The aim of...

    Alice Diterlizzi, Anna Tropea, ... Rosanna Apa in Archives of Gynecology and Obstetrics
    Article Open access 04 July 2024
  11. Homopolymer switches mediate adaptive mutability in mismatch repair-deficient colorectal cancer

    Mismatch repair (MMR)-deficient cancer evolves through the stepwise erosion of coding homopolymers in target genes. Curiously, the MMR genes MutS...

    Hamzeh Kayhanian, William Cross, ... Marnix Jansen in Nature Genetics
    Article Open access 03 July 2024
  12. A healthy live birth after mosaic blastocyst transfer in preimplantation genetic testing for GATA1-related cytopenia combined with HLA matching

    Background

    GATA1-related cytopenia (GRC) is characterized by thrombocytopaenia and/or anaemia ranging from mild to severe. Haematopoietic stem cell...

    Huiling Xu, Jiajie Pu, ... Xuemei Li in BMC Medical Genomics
    Article Open access 03 July 2024
  13. Respiratory infection- and asthma-prone, low vaccine responder children demonstrate distinct mononuclear cell DNA methylation pathways

    Background

    Infants with frequent viral and bacterial respiratory infections exhibit compromised immunity to routine immunizations. They are also more...

    David Martino, Nikki Schultz, ... Michael Pichichero in Clinical Epigenetics
    Article Open access 03 July 2024
  14. Screening primary carnitine deficiency in 10 million Chinese newborns: a systematic review and meta-analysis

    Background

    Primary carnitine deficiency (PCD) is a rare autosomal recessive fatty acid oxidation disorder caused by variants in SLC22A5, with its...

    **fu Zhou, Guilin Li, ... Liangpu Xu in Orphanet Journal of Rare Diseases
    Article Open access 03 July 2024
  15. Brown trout in Oder estuary tributaries: genetic structure, stocking, and admixture

    Several rivers that are tributaries of the Oder estuary are inhabited by Salmo trutta L, the most important of which are Ina, Gowienica, and...

    Rafał Bernaś, Anna Wąs-Barcz, ... Krzysztof Formicki in Journal of Applied Genetics
    Article Open access 03 July 2024
  16. Autism spectrum disorder in young patients with congenital central hypoventilation syndrome: role of the autonomic nervous system dysfunction

    Background

    Congenital central hypoventilation syndrome (CCHS) is a rare condition characterized by alveolar hypoventilation and autonomic nervous...

    Benjamin Dudoignon, Anna Maruani, ... Christophe Delclaux in Orphanet Journal of Rare Diseases
    Article Open access 03 July 2024
  17. Schlafen 11 further sensitizes BRCA-deficient cells to PARP inhibitors through single-strand DNA gap accumulation behind replication forks

    The preferential response to PARP inhibitors (PARPis) in BRCA-deficient and Schlafen 11 (SLFN11)-expressing ovarian cancers has been documented, yet...

    Hiroshi Onji, Sota Tate, ... Junko Murai in Oncogene
    Article Open access 03 July 2024
  18. Extent of magnitude representation deficit and relationship with arithmetic skills in children with 22q11.2DS

    Background

    Previous studies have produced conflicting results concerning the extent of magnitude representation deficit and its relationship with...

    Emilie Favre, Margot Piveteau, ... Caroline Demily in Orphanet Journal of Rare Diseases
    Article Open access 03 July 2024
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