Category GENE THERAPY RARE DISEASES

Novel Supramolecular CRISPR–Cas9 Carrier Enables More Efficient Genome Editing

Fifth generation polyrotaxane (PRX) carriers can effectively deliver CRISPR-Cas9 ribonucleoproteins (RNPs) The PRX carrier can cross the cell membrane, avoid endosomal action, and release Cas9 RNP for entry into the nucleusImage courtesy: Kumamoto University, The carriers, aminated polyrotaxanes, can flexibly and reversibly bind with Cas9 ribonucleoprotein and protect it from intracellular endosomal degradation CRISPR-Cas9 is

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Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A

BACKGROUND Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter. The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1–2 dose-escalation study. METHODS Was conducted an open-label, single-group, multicenter, phase 3

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Huge step forward towards gene therapy and diagnosis for genetic eye disease

New opportunities towards gene therapy and diagnosis for the blinding eye disease, retinal dystrophy, may now become available following work done by the Eye Genetics Research Unit at Children’s Medical Research Institute. This work was published in the Journal of Personalized Medicine today. The team looked at the RPGR gene which is involved in maintaining

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Gene Therapy for Thalassemia Ends Need for Transfusions in Young Children

Phase 3 clinical trial included children younger than 12 years Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients

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First gene therapy for Tay-Sachs disease successfully given to two children

Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years of development. Tay-Sachs is a severe neurological disease caused by a deficiency in an enzyme called HexA. This enzyme breaks down a fatlike substance that normally exists in very small, harmless amounts in the brain. Without HexA, however, this fatlike substance can accumulate

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Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome

Genethon, a unique non-profit gene therapy R&D organization founded by the French Muscular Dystrophy Association (AFM-Telethon), announced today that its lentiviral based gene therapy, developed in collaboration with French and British teams, has demonstrated long-term efficacy in eight patients with Wiskott-Aldrich syndrome, a rare and severe immune deficiency. “These results confirm the stability and good

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New gene therapy treatment resulted in sustained, clinically relevant benefits in children with Metachromatic Leucodystrophy: long term results published.

An investigational gene therapy showed benefit in pediatric patients with early-onset metachromatic leukodystrophy (MLD), a small study showed. Treatment with atidarsagene autotemcel (arsa-cel) preserved motor development and cognitive function in MLD patients enrolled in a prospective phase I/II trial or treated through expanded access programs, reported Alessandro Aiuti, MD, of the San Raffaele Telethon Institute for Gene

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Epilepsy Research Boosts Case for New Gene Therapy

Research from the School of Medicine suggests how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition. The gene therapy, developed by Stoke Therapeutics, is now in clinical trials. Because most Dravet syndrome cases are caused by a mutation in the SCN1A gene, resulting in

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Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss

Scientists at Boston Children’s Hospital have successfully used gene therapy to reverse a form of genetic hearing loss in mice. The team corrected a gene mutation that affects sensory hair cells in the inner ear, and the new jigsaw-like method could help improve gene therapy for other disorders. The target of the new treatment was

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