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Therapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors

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  • Post published:February 23, 2026
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Cancer is a major cause of human death worldwide. The vast majority of cancer patients suffer from solid tumors whose growth destroys vital organs. We propose to develop novel therapeutic…

Continue ReadingTherapeutic Opportunities To Target Tumor Initiating Cells in Solid Tumors

ZINC FINGER NUCLEASE-BASED STEM CELL THERAPY FOR AIDS

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  • Post published:February 23, 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

Stem cell based treatment strategy for Age-related Macular Degeneration (AMD)

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  • Post published:February 23, 2026
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Retinal degeneration represents a group of blinding diseases that are increasingly impacting the health and well being of Californians. It is estimated that by 2020, over 450,000 Californians will suffer…

Continue ReadingStem cell based treatment strategy for Age-related Macular Degeneration (AMD)

Development of Highly Active Anti-Leukemia Stem Cell Therapy (HALT)

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  • Post published:February 23, 2026
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Leukemias are cancers of the blood forming cells that afflict both children and adults. Many drugs have been developed to treat leukemias and related diseases. These drugs are often effective…

Continue ReadingDevelopment of Highly Active Anti-Leukemia Stem Cell Therapy (HALT)

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

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  • Post published:February 22, 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:February 22, 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:February 22, 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:February 22, 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:February 22, 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:February 22, 2026
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Continue ReadingMicroglia replacement therapy for CSF1R-related Leukoencephalopathy
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