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Stem Cell-Mediated Oncocidal Gene Therapy of Glioblastoma (GBM)

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  • Post published:May 12, 2026
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Brain tumors (BTs) are incurable, whether they start in the brain or spread there from other sites. Despite advances in surgical, radiation, pharmacologic, and gene therapies, survival with a BT…

Continue ReadingStem Cell-Mediated Oncocidal Gene Therapy of Glioblastoma (GBM)

Stem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials

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  • Post published:May 12, 2026
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Despite aggressive multimodal therapy and advances in imaging, surgical and radiation techniques, malignant brain tumors (high-grade gliomas) remain incurable, with survival often measured in months. Treatment failure is largely attributable…

Continue ReadingStem Cell-mediated Therapy for High-grade Glioma: Toward Phase I-II Clinical Trials

iPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa

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  • Post published:May 12, 2026
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Genetic skin diseases constitute a diverse group of several hundred diseases that affect up to 2% of the population and include common disease such as psoriasis, atopic dermatitis, and wound…

Continue ReadingiPS Cell-Based Treatment of Dominant Dystrophic Epidermolysis Bullosa

ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS

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  • Post published:May 12, 2026
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Some years ago it was discovered that patients homozygous for a natural mutation (the Δ32 mutation) in the CCR5 gene are generally resistant to HIV infection by blocking virus entry…

Continue ReadingZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS

A First-in-Class CIRSPR-CasX Gene Editing Therapy for Lowering Lp(a) to Prevent Cardiovascular Events

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  • Post published:May 11, 2026
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Continue ReadingA First-in-Class CIRSPR-CasX Gene Editing Therapy for Lowering Lp(a) to Prevent Cardiovascular Events

Gene Therapy for Alpha-1 Anti-Trypsin Deficiency

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  • Post published:May 11, 2026
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Continue ReadingGene Therapy for Alpha-1 Anti-Trypsin Deficiency

Late-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

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  • Post published:May 11, 2026
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Continue ReadingLate-stage development of AS-241, an UNC13A Targeting Antisense Oligonucleotide treatment for Amyotrophic Lateral Sclerosis, for IND-enabling studies

Advancement of a myotropic, liver-detargeting therapy for LGMD2i/R9

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  • Post published:May 11, 2026
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Continue ReadingAdvancement of a myotropic, liver-detargeting therapy for LGMD2i/R9

A first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia

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  • Post published:May 11, 2026
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Continue ReadingA first-in-class CRISPR-CasX gene editor silencing APOC3 transcription for the treatment of Severe Hypertriglyceridemia

Microglia replacement therapy for CSF1R-related Leukoencephalopathy

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  • Post published:May 11, 2026
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Continue ReadingMicroglia replacement therapy for CSF1R-related Leukoencephalopathy
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