Gene Therapy
- November 13, 2019
- 0
- Category:
- Tags: antigensbiologic therapyCHILDChildhoodchildrenendpointFDAGene TherapyGenomic medicineGenomicshealth care quality/access/evaluationkidsmedicine (genomic)meetsmuscular atrophiesMuscular AtrophyPediatricspharmacokineticPrecision MedicineprimaryRisdiplamSMAsocial determinants of healthspinal muscle atrophySpinal Muscular Atrophysunfishu.s. food and drug administrationunited states food and drug administration
Risdiplam, an oral investigational agent designed to increase and sustain SMN protein levels of patients with spinal muscular atrophy (SMA), met the primary endpoint in the pivotal part 2 of...
Read more- October 22, 2019
- 0
- Category:
- Tags: AcademyBeverlyCell TherapychildrenDavidsonElectedGene TherapygeneticsHereditary DiseasesmedicineNationalNational Academy of Medicineneurobiologyneurodegenerative brain disorderNewswisePediatricsPhD
Beverly L. Davidson, PhD, director of the Raymond G. Perelman Center for Cellular and Molecular Therapeutics at Children's Hospital of Philadelphia, has been elected to the National Academy of Medicine....
Read more- September 23, 2019
- 0
- Category:
- Tags: CellCell CulturecheaperfasterGeneGene TherapyGene TransfergenesGeneticGenomeglutamineheartImprovedinflammationLiving CellsmedicineMuscular AtrophyplasmidproductionresearchSpinal Muscular AtrophytherapyToolvirus
The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new study, published on 19 September...
Read more- September 12, 2019
- 0
- Category:
- Tags: bloodbrainCelldevelopDisabilityGeneGene Therapygeneshelpsischemic strokeLaboratoryNerveNeural stem cellsneuronspatientsProgenitor CellsStem Cellsstroketherapy
Ischemic stroke is a condition in which parts of the brain lose their blood supply, causing nerve damage. Once brain tissue suffers irreparable harm, the patient will experience irreversible disability...
Read more- September 10, 2019
- 0
- Category:
- Tags: CellDNAGenegene editingGene TherapyGeneeditingGeneticGenomeGenome EditingnanocapsulesNanoparticlesnanotechnologypackedplatformPromisingresearchtherapiestools
New tools for editing genetic code offer hope for new treatments for inherited diseases, some cancers, and even stubborn viral infections. But the typical method for delivering gene therapies to...
Read more- August 8, 2019
- 0
- Category:
- Tags: breakthroughcancerCAR-TCoverGeneGene TherapyLeukemiaLymphomaMedicaretherapy
Studies have shown that CAR-T is effective against certain types of cancers, but can cause severe side effects. Source link
Read more- August 7, 2019
- 0
- Category:
- Tags: CRISPRcysticcystic fibrosiseditingFibrosisGeneGene TherapyGeneticGenomeGenome EditingMutationopensOrganoidsPerspectivesresearchResearch Projectsplicingstrategytreatment
The approach adopted by the team of the University of Trento, led by Anna Cereseto, opens new perspectives in the treatment of cystic fibrosis, a genetic disease for which no...
Read more- July 30, 2019
- 0
- Category:
- Tags: All Journal NewsBiomechanicsCell biologychildrenCHOPFootprintGeneGene TherapygeneticsNature (journal)NewswisePersonalized MedicineRedefinesresearchTeamtherapyvectorviral
Building on a track record of developing adeno-associated viral (AAV) vectors as a groundbreaking clinical tool for gene therapy and gene editing, Children's Hospital of Philadelphia (CHOP) researchers report a...
Read more- July 18, 2019
- 0
- Category:
- Tags: CellCell nucleuscrucialCytoplasmdevelopmentfunctionGeneGene TherapygenesHypoxiaInsightmicroRNAresearchRNARNA interferencetherapy
Scientists at the University of Eastern Finland and the University of Oxford have shown that small RNA molecules occurring naturally in cells, i.e. microRNAs, are also abundant in cell nuclei....
Read more- June 20, 2019
- 0
- Category:
- Tags: ApplyingbiologyBotherCell culturesdrug discoveryDrug licensingEuropeGene TherapymeasurementMedicine and health sciencesNatural materialsReproducibilityscience
by Carmen H. Coxon, Colin Longstaff, Chris Burns Both basic and translational research are continuously evolving, but the principles that underpin research integrity remain constant. These include rational, hypothesis-driven, and...
Read more- June 19, 2019
- 0
- Category:
- Tags: anemiabloodCellchildrenClinical TrialdiseaseGeneGene TherapyGeneticHematologyLife ExpectancypainProgressrealRed Blood CellsSicklesickle cell diseasesignsSudden Cardiac Death
A decade ago, the United Nations declared June 19 to be World Sickle Cell Awareness Day- and for good reason. Source link
Read more- June 19, 2019
- 0
- Category:
- Tags: Babies039bloodBlood Vesselblood vesselsBluecancerCapillariesCas9childrenCRISPRCritical CaredisorderDNADysplasiaEfficacyFatalGeneGene TherapygenesGeneticHospitalLaboratorylipslungsmedicineMutationNanoparticleNanoparticlesNewbornOXYGENpinpointproliferationproteinrespiratoryScientistsskinTurns
Scientists used a gene editing method called CRISPR/Cas9 to generate mice that faithfully mimic a fatal respiratory disorder in newborn infants that turns their lips and skin blue. The new...
Read more- June 13, 2019
- 0
- Category:
- Tags: anemiabloodboneBone MarrowCellchildrendiseaseeducationGeneGene TherapyHematologyHemoglobinmortalityMutationneededOXYGENPenicillinprophylaxisproteinpublic healthRed Blood CellsresearchscreeningSickleSickle Cell Anemiasickle cell diseaseThalassemiatreatments
The promise of new treatments for sickle cell disease, a group of inherited red blood cell disorders, has never been so great, but it will only be realized if there...
Read more- June 5, 2019
- 0
- Category:
- Tags: boneBone MarrowcarryCellCRISPReditingGenegene editingGene TherapygenesGeneticGenomein vivomuscleMutationPetri DishresearchScientistsskinStem CellsSuccessfullyVivo
Scientists at Harvard University have successfully proven that stem cells can be edited inside the body, rather than in a petri dish. Source link
Read more- May 28, 2019
- 0
- Category:
- Tags: AIDSbloodboneBone MarrowcancerCas9CellCRISPRdisordersDNAeditGenegene editingGene TherapygenesGeneticgeneticsGenomeGoldGold NanoparticlesHIVImmune SystemimmunologyInfectious DiseasesinheritedmedicineNanoparticleNanoparticlesNucleaseproteinresearchScientiststiedvirus
Scientists at Fred Hutchinson Cancer Research Center took a step toward making gene therapy more practical by simplifying the way gene-editing instructions are delivered to cells. Source link
Read more- May 25, 2019
- 0
- Category:
- Tags: ApprovedbasedchildrenClinical TrialdeliveryDiscoveredExpertGeneGene TherapygenesGeneticgeneticsHospitalmedicinemuscleMuscle AtrophyMuscular AtrophyMutationnewlyPediatricsPennpharmaceuticalsproteinRare DiseaseresearchSMASpinal Muscular AtrophytherapyvehiclevirusViruses
James Wilson, MD, PhD, recalls being struck by the devastating toll of rare diseases as a young physician in the 1980s. He set out on a path to correct the...
Read more- May 7, 2019
- 0
- Category:
- Tags: AllergyAnschutzcancerCellCell biologychildrenCuCytomegalovirusdiseaseeczemaGeneGene TherapyGeneticIdentifyImmunodeficiencyimmunologyinflammationInflammatory Bowel DiseaseLung DiseasemedicineMicrobiologyMutationPediatricsresearchResearchers
[ad_1] A new immunodeficiency disease caused by a novel genetic mutation has been identified by researchers at the University of Colorado Anschutz Medical Campus providing unique insights into cell biology....
Read more- May 3, 2019
- 0
- Category:
- Tags: agentALSamyotrophic lateral sclerosisdrug developmentexperimentalFormGene TherapyGeneticpromiseshowsSlowing
Researchers successfully target elevated levels of a harmful protein, offering hope to people with a genetic form of amyotrophic lateral sclerosis. Medscape Medical News Source link
Read more- May 1, 2019
- 0
- Category:
- Tags: challengeDNA repairEmbryosGeneGene TherapyGermlineHealth economicsHIV preventionHuman Geneticspeer reviewregulatingScientiststherapy
by Arthur Caplan The announcement of He Jiankui’s germline editing of human embryos has been followed by a torrent of almost universal criticism of the claim on scientific and ethical...
Read more- April 18, 2019
- 0
- Category:
- Tags: 039BubbleautosomalbabiesbacteriabloodboneBone MarrowBoy039CellchemotherapychildrenchromosomeClinical TrialCuresdiarrheadiseaseFatalGeneGene TherapyGeneticGenetic DisorderHospitalImmune SystemImmunodeficiencyInheritLeukemiamedicineMutationSevere Combined ImmunodeficiencyskintherapyThrushVirusesX chromosomeX-linked
[ad_1] A new gene therapy created by US researchers appears to have cured eight infant boys born with a rare genetic disorder referred to as “Bubble Boy” disease. Source link...
Read more