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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
Ex vivo dual gene therapy using human adipocytes secreting anti-HER2 antibody on HER2-positive xenograft tumor models
CONCLUSION: Ex vivo gene-transduced adipocytes might be useful for cell-based gene therapy. This system may be a platform for various antibody therapies.PMID:37612442 | DOI:10.1007/s12282-023-01497-8
Source: Breast Cancer - August 23, 2023 Category: Cancer & Oncology Authors: Ryotaro Teranaka Hiroshi Fujimoto Takahito Masuda Masayuki Kuroda Yasuyuki Aoyagi Takeshi Nagashima Mamoru Takada Junta Sakakibara Hideyuki Yamada Hiroto Yamamoto Yoshitaka Kubota Masayuki Ohtsuka Source Type: research
Treatment of Inherited Platelet Disorders: Current Status and Future Options
Hamostaseologie. 2023 Aug;43(4):261-270. doi: 10.1055/a-2080-6602. Epub 2023 Aug 23.ABSTRACTInherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 (www.awmf.org) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is giv...
Source: Hamostaseologie - August 23, 2023 Category: Hematology Authors: Caroline Bargehr Ralf Kn öfler Werner Streif Source Type: research
Ex vivo dual gene therapy using human adipocytes secreting anti-HER2 antibody on HER2-positive xenograft tumor models
CONCLUSION: Ex vivo gene-transduced adipocytes might be useful for cell-based gene therapy. This system may be a platform for various antibody therapies.PMID:37612442 | DOI:10.1007/s12282-023-01497-8
Source: Breast Cancer - August 23, 2023 Category: Cancer & Oncology Authors: Ryotaro Teranaka Hiroshi Fujimoto Takahito Masuda Masayuki Kuroda Yasuyuki Aoyagi Takeshi Nagashima Mamoru Takada Junta Sakakibara Hideyuki Yamada Hiroto Yamamoto Yoshitaka Kubota Masayuki Ohtsuka Source Type: research
Treatment of Inherited Platelet Disorders: Current Status and Future Options
Hamostaseologie. 2023 Aug;43(4):261-270. doi: 10.1055/a-2080-6602. Epub 2023 Aug 23.ABSTRACTInherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 (www.awmf.org) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is giv...
Source: Hamostaseologie - August 23, 2023 Category: Hematology Authors: Caroline Bargehr Ralf Kn öfler Werner Streif Source Type: research
Ex vivo dual gene therapy using human adipocytes secreting anti-HER2 antibody on HER2-positive xenograft tumor models
CONCLUSION: Ex vivo gene-transduced adipocytes might be useful for cell-based gene therapy. This system may be a platform for various antibody therapies.PMID:37612442 | DOI:10.1007/s12282-023-01497-8
Source: Breast Cancer - August 23, 2023 Category: Cancer & Oncology Authors: Ryotaro Teranaka Hiroshi Fujimoto Takahito Masuda Masayuki Kuroda Yasuyuki Aoyagi Takeshi Nagashima Mamoru Takada Junta Sakakibara Hideyuki Yamada Hiroto Yamamoto Yoshitaka Kubota Masayuki Ohtsuka Source Type: research
Treatment of Inherited Platelet Disorders: Current Status and Future Options
Hamostaseologie. 2023 Aug;43(4):261-270. doi: 10.1055/a-2080-6602. Epub 2023 Aug 23.ABSTRACTInherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 (www.awmf.org) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is giv...
Source: Hamostaseologie - August 23, 2023 Category: Hematology Authors: Caroline Bargehr Ralf Kn öfler Werner Streif Source Type: research
Liposomal delivery of gene therapy for ovarian cancer: a systematic review
CONCLUSION: Liposomal delivery of gene therapy for ovarian cancer shows promise in many in vivo studies. Emerging polymer-coated and ligand-targeted liposomes have been gaining interest as they have been shown to have more stability and specificity. We found that gene therapy involving microRNAs was the most frequently studied. Overall, liposomal genetic therapy has been shown to reduce tumor size and weight and improve survivability. More research involving the delivery and targets of gene therapy for ovarian cancer may be a promising avenue to improve patient outcomes.PMID:37612696 | DOI:10.1186/s12958-023-01125-2
Source: Reproductive Biology - August 23, 2023 Category: Reproduction Medicine Authors: Jin Sung Son Ryan Chow Helena Kim Toney Lieu Maria Xiao Sunny Kim Kathy Matuszewska Madison Pereira David Le Nguyen Jim Petrik Source Type: research
Treatment of Inherited Platelet Disorders: Current Status and Future Options
Hamostaseologie. 2023 Aug;43(4):261-270. doi: 10.1055/a-2080-6602. Epub 2023 Aug 23.ABSTRACTInherited platelet disorders (IPDs) comprise a heterogeneous group of entities that manifest with variable bleeding tendencies. For successful treatment, the underlying platelet disorder, bleeding severity and location, age, and sex must be considered in the broader clinical context. Previous information from the AWMF S2K guideline #086-004 (www.awmf.org) is evaluated for validity and supplemented by information of new available and future treatment options and clinical scenarios that need specific measures. Special attention is giv...
Source: Hamostaseologie - August 23, 2023 Category: Hematology Authors: Caroline Bargehr Ralf Kn öfler Werner Streif Source Type: research
Ex vivo dual gene therapy using human adipocytes secreting anti-HER2 antibody on HER2-positive xenograft tumor models
CONCLUSION: Ex vivo gene-transduced adipocytes might be useful for cell-based gene therapy. This system may be a platform for various antibody therapies.PMID:37612442 | DOI:10.1007/s12282-023-01497-8
Source: Breast Cancer - August 23, 2023 Category: Cancer & Oncology Authors: Ryotaro Teranaka Hiroshi Fujimoto Takahito Masuda Masayuki Kuroda Yasuyuki Aoyagi Takeshi Nagashima Mamoru Takada Junta Sakakibara Hideyuki Yamada Hiroto Yamamoto Yoshitaka Kubota Masayuki Ohtsuka Source Type: research
