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A shift in focus towards precision oncology, driven by revolutionary nanodiagnostics; revealing mysterious pathways in colorectal carcinogenesis
This article examines current nanomedicine patents for CRC, including the work of Delta-Fly, Merrimack, and Pfenning, Meaning & Partner, among others. In terms of future nanomedicine research and development, ligand production, particle size, and clearance are crucial factors. Lastly, the numerous nanostructures utilized in nanomedicine for targeted drug administration and diagnostics indicate optimistic prospects for enhancing CRC treatment. The successes of nanomedicine research and development for existing colon cancer treatments are also highlighted in this review.PMID:37650995 | DOI:10.1007/s00432-023-05331-8
Source: Clinical Colorectal Cancer - August 31, 2023 Category: Cancer & Oncology Authors: Satyam Sharma Sankha Bhattacharya Kajal Joshi Sanjiv Singh Source Type: research

Scale-Up of Protein Purification: Downstream Processing Issues
Methods Mol Biol. 2023;2699:61-75. doi: 10.1007/978-1-0716-3362-5_5.ABSTRACTLarge-scale chromatography operations continue to occupy the central position in the overall strategy for downstream processing and purification of therapeutic protein products for human use. As the biopharmaceutical industry looks forward to embracing new therapeutic modalities such as viral vector-mediated gene therapy, it is becoming evident that chromatographic separations will be also be crucial for success in that discipline. The current industry focus on cell culture intensification strategies that can result in increased process efficiency ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: John Joseph Milne Source Type: research

Chromatographic Purification of Viral Vectors for Gene Therapy Applications
Methods Mol Biol. 2023;2699:51-60. doi: 10.1007/978-1-0716-3362-5_4.ABSTRACTChromatography has been a mainstay in the downstream processing and purification of biopharmaceutical medicines. Until now, this has largely involved the purification of protein products such as recombinant enzymes and monoclonal antibodies using large-scale column chromatography methods. The development of advanced therapeutic medicinal products (ATMP) is heralding in a new era of therapeutics for a range of indications. These new therapeutics use diverse substances ranging from live stem cell preparations to fragments of nucleic acid enclosed in ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: Aoife Mair éad Kearney Source Type: research

Scale-Up of Protein Purification: Downstream Processing Issues
Methods Mol Biol. 2023;2699:61-75. doi: 10.1007/978-1-0716-3362-5_5.ABSTRACTLarge-scale chromatography operations continue to occupy the central position in the overall strategy for downstream processing and purification of therapeutic protein products for human use. As the biopharmaceutical industry looks forward to embracing new therapeutic modalities such as viral vector-mediated gene therapy, it is becoming evident that chromatographic separations will be also be crucial for success in that discipline. The current industry focus on cell culture intensification strategies that can result in increased process efficiency ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: John Joseph Milne Source Type: research

Chromatographic Purification of Viral Vectors for Gene Therapy Applications
Methods Mol Biol. 2023;2699:51-60. doi: 10.1007/978-1-0716-3362-5_4.ABSTRACTChromatography has been a mainstay in the downstream processing and purification of biopharmaceutical medicines. Until now, this has largely involved the purification of protein products such as recombinant enzymes and monoclonal antibodies using large-scale column chromatography methods. The development of advanced therapeutic medicinal products (ATMP) is heralding in a new era of therapeutics for a range of indications. These new therapeutics use diverse substances ranging from live stem cell preparations to fragments of nucleic acid enclosed in ...
Source: Mol Biol Cell - August 30, 2023 Category: Molecular Biology Authors: Aoife Mair éad Kearney Source Type: research

A Comprehensive Review on Current Treatments and Challenges Involved in the Treatment of Ovarian Cancer
In conclusion, tailored nanocarriers would optimize medication delivery into the intracellular compartment before optimizing intra-tumour distribution. Other novel treatment possibilities for ovarian cancer include tumour vaccines, gene therapy, targeting epigenetic alteration, and biologically targeted compounds. These characteristics might enhance the therapeutic efficacy.PMID:37642226 | DOI:10.2174/1568009623666230811093139
Source: Current Cancer Drug Targets - August 29, 2023 Category: Cancer & Oncology Authors: Saika Saman Nimisha Srivastava Mohd Yasir Iti Chauhan Source Type: research

Systemic gene therapy rescues retinal dysfunction and hearing loss in a model of Norrie disease
EMBO Mol Med. 2023 Aug 29:e17393. doi: 10.15252/emmm.202317393. Online ahead of print.ABSTRACTDeafness affects 5% of the world's population, yet there is a lack of treatments to prevent hearing loss due to genetic causes. Norrie disease is a recessive X-linked disorder, caused by NDP gene mutation. It manifests as blindness at birth and progressive sensorineural hearing loss, leading to debilitating dual sensory deprivation. To develop a gene therapy, we used a Norrie disease mouse model (Ndptm1Wbrg ), which recapitulates abnormal retinal vascularisation and progressive hearing loss. We delivered human NDP cDNA by intraven...
Source: Molecular Medicine - August 29, 2023 Category: Molecular Biology Authors: Valda Pauzuolyte Aara Patel James R Wawrzynski Neil J Ingham Yeh Chwan Leong Rajvinder Karda Maria Bitner-Glindzicz Wolfgang Berger Simon N Waddington Karen P Steel Jane C Sowden Source Type: research

Current avenues of gene therapy in Pompe disease
Curr Opin Neurol. 2023 Jul 20. doi: 10.1097/WCO.0000000000001187. Online ahead of print.ABSTRACTPURPOSE OF THE REVIEW: Pompe disease is a rare, inherited, devastating condition that causes progressive weakness, cardiomyopathy and neuromotor disease due to the accumulation of glycogen in striated and smooth muscle, as well as neurons. While enzyme replacement therapy has dramatically changed the outcome of patients with the disease, this strategy has several limitations. Gene therapy in Pompe disease constitutes an attractive approach due to the multisystem aspects of the disease and need to address the central nervous syst...
Source: Epilepsy Curr - August 28, 2023 Category: Neurology Authors: Carmen Leon-Astudillo Prasad D Trivedi Ramon C Sun Matthew Gentry Barry J Byrne Manuela Corti Source Type: research

Fight Aging! Newsletter, August 28th 2023
In conclusion, we identified 20 genes with significant evolutionary signals unique to long-lived species, which provided new insight into the lifespan extension of mammals and might bring new strategies to extend human lifespan. « Back to Top Trials of Xenotransplantation of Pig Organs into Humans Continue https://www.fightaging.org/archives/2023/08/trials-of-xenotransplantation-of-pig-organs-into-humans-continue/ Researchers have genetically engineered pigs to overcome the known barriers to transplantation of pig organs into humans, and have reached the stage of conducting transplants in...
Source: Fight Aging! - August 27, 2023 Category: Research Authors: Reason Tags: Newsletters Source Type: blogs

Sickle Cell Disease: From Genetics to Curative Approaches
Annu Rev Genomics Hum Genet. 2023 Aug 25;24:255-275. doi: 10.1146/annurev-genom-120122-081037.ABSTRACTSickle cell disease (SCD) is a monogenic blood disease caused by a point mutation in the gene coding for β-globin. The abnormal hemoglobin [sickle hemoglobin (HbS)] polymerizes under low-oxygen conditions and causes red blood cells to sickle. The clinical presentation varies from very severe (with acute pain, chronic pain, and early mortality) to normal (few complications and a normal life span). The variability of SCD might be due (in part) to various genetic modulators. First, we review the main genetic factors, polymor...
Source: Annual Review of Genomics and Human Genetics - August 25, 2023 Category: Genetics & Stem Cells Authors: Giulia Hardouin Elisa Magrin Alice Corsia Marina Cavazzana Annarita Miccio Michaela Semeraro Source Type: research