Gene therapy offers hope for patients with hearing loss

Gene therapy offers hope for patients with hearing loss

Hearing loss is one of the most prevalent disorders and it is estimated that over one billion people suffer from hearing loss and approximately one-two children in every 1,000 births are born with congenital hearing loss. It is not therefore surprising that screening for newborns is an essential component of newborn screening programmes. Hearing …

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Could gene therapy cure PWS?

Could gene therapy cure PWS?

Understanding the relationship between thyroid health and Prader-Willi Syndrome (PWS) can shed light on the intricate connections between hormonal imbalances and genetic disorders. Both play crucial roles in regulating bodily functions, and their interaction can significantly impact an individual’s well-being. Hypothyroidism, which involves an underactive thyroid gland, can further slow down metabolism and contribute to weight gain, which …

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Scientists transform skin cells into functional eggs in mice

Scientists transform skin cells into functional eggs in mice

New research from Oregon Health & Science University describes the science behind a promising technique to treat infertility by turning a skin cell into an egg that is capable of producing viable embryos. Researchers at OHSU documented in vitro gametogenesis, or IVG, in a mouse model through the preliminary steps of a technique that relies …

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Gene therapy improves advanced heart failure in animal model: Study | Health

Gene therapy improves advanced heart failure in animal model: Study | Health

During a heart attack, blood stops flowing into the heart. Without oxygen, a portion of the heart muscle dies. Gene therapy improves advanced heart failure in animal model: Study(Image by InspiredImages from Pixabay ) {{^userSubscribed}} {{/userSubscribed}} {{^userSubscribed}} {{/userSubscribed}} The heart muscle does not regenerate, instead, it replaces dead tissue with a scar made of cells …

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Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy

Excision Bio Seeks to Suppress HIV Replication With CRISPR Gene Therapy

CGTLive™ first published this article. This version has been lightly edited. A cure for HIV-1 becomes more feasible as advances continue in gene therapy research and more trials open investigations on potential novel treatments. One such approach among the frontrunners developing a novel therapy for treating HIV-1 is Excision BioTherapeutics’ EBT-101. EBT-101 uses the CRISPR/Cas9 …

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More sickle cell patients might benefit from bone marrow transplant

More sickle cell patients might benefit from bone marrow transplant

SAN DIEGO — The sickle cell community has for the past few days been buzzing with news of the first-ever approved gene therapies for the devastating disease. Meanwhile, researchers at the American Society of Hematology meeting on Tuesday are reporting advances in a less expensive and more established strategy proven to cure patients: bone marrow …

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Novel mechanism leads to motor neuron degeneration in spinal muscular atrophy

Novel mechanism leads to motor neuron degeneration in spinal muscular atrophy

The lab of Yongchao C. Ma, PhD, at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago uncovered a novel mechanism that leads to motor neuron degeneration in spinal muscular atrophy (SMA). This discovery offers a new target for treatment that overcomes important limitations of gene therapy and other …

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Way to deliver clinically used gene therapy vectors: Study | Health

Way to deliver clinically used gene therapy vectors: Study | Health

Researchers examined the safety and practicality of gene delivery to several brain regions in animals using a noninvasive, ultrasound-based approach, and their findings imply that when more locations are opened, gene delivery effectiveness improves within each targeted site. Way to deliver clinically used gene therapy vectors: Study(Shutterstock) {{^userSubscribed}} {{/userSubscribed}} {{^userSubscribed}} {{/userSubscribed}} Shirin Nouraein, a PhD …

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