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New hope for those living with Sickle Cell Disease thanks to experimental gene treatment
08 Jun 2024 09:36 GMT
…
The treatment is a gene cell therapy recently approved by the … regular things like hold cups or walk,” Gray said.
She … the first cell-based gene therapies that target the … treatment, whether with experimental therapy or for other care management procedures …
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Gene therapy restores hearing in deaf children
07 Jun 2024 14:17 GMT
… added benefits of the gene therapy when administrated to both … events, none were serious or dose-limiting. All five … conducted the first-ever gene therapy trial for DFNB9, treating … the development of scalable gene therapy platforms to facilitate future …
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Cencora VP & Head of Cell & Gene Therapy Service Line Discusses Industry Pain Points and Technological Capabilities
07 Jun 2024 12:31 GMT
PC: Could you describe some common pain points in the space that the industry has been experiencing?
Cheng: I will build off of what I just mentioned. If you think about patient access, if you think about the regulatory requirements, if you think about …
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Gene therapy trial: restored hearing in children with hereditary deafness
07 Jun 2024 11:13 GMT
… deafness 9 (DFNB9).
Study: Bilateral gene therapy in children with autosomal recessive … of substance abuse, complex immunodeficiency or organ transplantation, a history of … neurological or psychiatric disorders, and a history …
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Would you want to know if you're likely to get cancer or dementia? Here's what people who've had their results say
08 Jun 2024 01:06 GMT
… diagnosed or died, you are referred through the NHS genetics service … surgery and multiple rounds of therapy and drugs, says: … to get tested for PSEN1 gene mutations, associated with familial … senior responsible officer for NHS genomics, said: "It can …
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First-ever gene therapy restores hearing in both ears of children born deaf
07 Jun 2024 02:35 GMT
… deafness. Unfortunately, no pharmaceutical drugs or treatments are available today to … deafness.
However, in recent years, gene therapy has emerged as a potential … in Boston, Massachusetts.
This innovative gene therapy experiment has shown promise in …
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Gene therapy restores hearing to children with inherited deafness
07 Jun 2024 02:23 GMT
… shows added benefits of the gene therapy when administrated to both … were observed over a 13 or 26-week period and showed … were no dose-limiting toxicity or serious adverse events.
The trial … their results show that the gene therapy is “feasible, safe, and …
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Comparative analysis of multiplexed in situ gene expression profiling technologies
07 Jun 2024 21:53 GMT
… brain sections (including coronal or sagittal sections), generated … converted into fluorescent signals, or both. All datasets exhibited … or when thresholding molecular counts for any individual gene … gene panel. We downloaded the dataset from the 10x Genomics …
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CRISPR Therapy Warrants Further Study for Inherited Retinal Diseases
07 Jun 2024 19:04 GMT
… research demonstrates that CRISPR gene therapy for inherited vision loss … PhD, director, Ocular Genomics Institute and Berman-Gund Laboratory … by a homozygous or compound heterozygous IVS26 variant … related to treatment or procedure, or any dose-limiting …
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Gene Therapy R&D market is projected to grow at a CAGR of 29.8% by 2034: Visiongain
06 Jun 2024 16:41 GMT
… therapeutic applications of gene therapy. As the industry matures, gene therapy stands to revolutionize … patients who previously faced limited or no treatment options. Download Exclusive … trials, with many studies delayed or halted due to lockdowns, travel …