Category Archives: Cell Therapy

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Abstract Graphical Abstract

Long-Term Follow-Up of the First In Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 For Severe Hemophilia B

Scientists present longitudinal follow up data of subjects who participated in the first trial of a systemically-delivered adeno-associated viral (AAV) vector. Adult males with severe hemophilia B received an AAV2 vector at doses ranging from 8×10¹⁰ – 2×10¹² vg/kg to target hepatocyte-specific expression of coagulation factor IX; a subset were followed for 12-15 years post vector administration.

[Molecular Therapy]

Abstract Graphical Abstract

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Publication

Efficient CRISPR/Cas9-Mediated Gene Ablation in Human Keratinocytes to Recapitulate Genodermatoses: Modeling of Netherton Syndrome

Researchers have established a highly efficient method for precise deletion of critical gene sequences in primary human keratinocytes, based on CRISPR/Cas9-mediated gene editing.

[Molecular Therapy-Methods & Clinical Development]

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Publication

Phase I Trial of Expanded, Activated Autologous NK Cell Infusions with Trastuzumab in Patients with HER2-Positive Cancers

The authors infused expanded, activated autologous natural killer cells to potentiate trastuzumab-mediated antibody-dependent cell cytotoxicity in patients with HER2-positive malignancies.

[Gene Therapy]

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Lentiviral Gene Therapy Rescues p47^phox Chronic Granulomatous Disease and the Ability to Fight Salmonella Infection in Mice

Investigators tested the ability of a gene therapy approach to control infection by challenging p47^phox-null mice with Salmonella Typhimurium, a leading cause of sepsis in chronic granulomatous diseasep atients, and found that mice reconstituted with lentivirus-transduced hematopoietic stem cells had a reduced bacterial load compared with untreated mice.

[Gene Therapy]

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Analysis of Aflibercept Expression in Non-Human Primates following Intravitreal Administration of ADVM-022, a Potential Gene Therapy for Neovascular Age-Related Macular Degeneration

Researchers explored whether ADVM-022, a recombinant adeno-associated virus vector designed to express aflibercept, could induce anti-VEGF protein levels comparable to those observed following a single bolus intravitreal injection of the standard-of-care aflibercept recombinant protein.

[Molecular Therapy-Methods & Clinical Development]

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Neural Stem Cell Delivery via Porous Collagen Scaffolds Promotes Neuronal Differentiation and Locomotion Recovery in Spinal Cord Injury

Scientists demonstrated that grafts based on porous collagen-based scaffolds, similar to biomaterials utilized clinically in induced regeneration, could deliver and protect embryonic neural stem cells at spinal cord injury (SCI) sites, leading to significant improvement in locomotion recovery in an experimental mouse SCI model.

[NPJ Regenerative Medicine]

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Novel Delivery of Cellular Therapy to Reduce Ischemia Reperfusion Injury in Kidney Transplantation

The authors investigated the potential capability of multipotent adult progenitor cells in kidney normothermic machine perfusion.

[American Journal of Transplantation]

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Comparison of the Regenerative Effects of Bone Marrow/Adipose-Derived Stem Cells in the Asherman Model following Local or Systemic Administration

Scientists compared the effects of systemic or local intrauterine injection of bone marrow or adipose-derived mesenchymal stem cells on the endometrium in a rat model of Asherman’s syndrome.

[Journal of Assisted Reproduction and Genetics]

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review

Advances in the Development of Gene Therapy, Noncoding RNA, and Exosome‐Based Treatments for Tendinopathy

The authors summarize the current understanding and treatments for tendinopathy. They also highlight recent advances in gene therapy, the potential of noncoding RNAs, such as microRNAs, and exosomes, which are nanometer‐sized extracellular vesicles secreted from cells, for the treatment of tendinopathy.

[Annals of the New York Academy of Sciences]

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Recovery of Motricity and Micturition after Transplantation of Mesenchymal Stem Cells in Rats Subjected to Spinal Cord Injury

Investigators evaluated the effect of human adipose-derived stem cell infusion on impaired hindlimb function and urinary continence after spinal cord contusion in rats.

[Neuroscience Letters]