POSTS TAGGED AS gene therapy

Successful Gene Therapy for Congenital Deafness

A medical first- Gene therapy allows boy to hear
“An 11-year-old boy recently became the first person in the U.S. to [MORE]

An RNA Switch for Modulating Gene Expression

An RNA switch for modulating gene expression
“The RNA-based switch controls mammalian gene expression through modulation of a synthetic polyA signal [MORE]

Lipid Nanoparticle Delivery of Anti-Inflammation siRNAs

Lipid nanoparticle delivery of anti-inflammation siRNAs
“Here, [from MIT researchers] several lipid nanoparticle (LNP) formulations are examined, and a lead candidate [MORE]

Gene Therapy in the Brain

Gene therapy can repair neural connections in the brain
“[University of Minnesota] researchers evaluated a new form of gene therapy invented [MORE]

MicroRNA-22 Gene Therapy for Liver Cancer

miR-22 gene therapy treats HCC by promoting anti-tumor immunity and enhancing metabolism
“Our [researchers at the University of California Davis] data [MORE]

One Shot Gene Therapy for Sickle Cell Disease

One Shot Gene Therapy for Sickle Cell Disease
“On July 27 in Science, scientists from Children’s Hospital of Philadelphia and the [MORE]

How Patient Age, Vector Dose, and Immune Response Figure in Gene Therapy for Duchenne Muscular Dystrophy

Gene editing therapy for DMD
“Based on the finding, the researchers [Sarepta Therapeutics] identified a need for more data on the [MORE]

Gene Therapy for Glaucoma

Glaucoma
“The major risk factor for glaucoma is elevated intraocular pressure, which is regulated by the balance between the secretion of [MORE]

Gene Therapy for Aging Hearts

Anti-aging gene shown to rewind heart age by 10 years
“Associated with exceptional longevity, carriers of healthy mutant genes, like those [MORE]

Gene Therapy for Rare Form of SCID

Lentiviral Gene Therapy for Artemis-Deficient SCID
“The DNA-repair enzyme Artemis is essential for rearrangement of T- and B-cell receptors. Mutations in [MORE]

2022’s Medical Milestones

Global effort realizes first 100% complete sequence of human genome
“The Human Genome Project has been running for decades, famously publishing [MORE]

Gender Modeling of Sex Differences

Modeling sex differences in humans using isogenic induced pluripotent stem cells
“In this work we [at Hebrew University, Jerusalem] developed a [MORE]

Gel-Delivered Topical Gene Therapy

Five-year-old Rowan Millner has dystrophic epidermolysis bullosa
From Stanford University:  “People with recessive dystrophic epidermolysis bullosa have a genetic mutation that [MORE]

Base-Editing Gene Therapy Cures a Child’s Incurable Leukemia

Teenager’s “incurable” leukemia cured by new base editing gene therapy
“A teenager in the UK has become the first person to [MORE]