Not HSV related, but the gene editing trial against transfusion-dependent beta thalassemia (βTDTβ), as well as those with sickle cell disease (βSCDβ), continues to pile up good data in phase1/2. There is now data from 22 patients.
Note this is an ex-vivo treatment using CRISPR targeting human cells. FHC are working on an in-vivo treatment using meganucleases targeting a latent virus. So there are important differences here.
But it's still nice that gene editing is showing good results here.
https://www.yahoo.com/finance/news/crispr-crsp-positive-gene-therapy-145002816.html
**"**The gene-editing therapy candidate demonstrated a consistent and sustained response to treatment in the given patient population.
Notably, a single dose of CTX001 is being evaluated in the phase I/II CLIMB THAL-111 study for treating TDT, and in the phase I/II CLIMB SCD-121 study for severe SCD. New data from the studies showed that treatment with CTX001 resulted in all 15 patients with TDT remaining transfusion independent at the last follow-up. Meanwhile, all seven patients with SCD were free of vaso-occlusive crises through last follow-up.
Importantly, five patients with TDT and two with SCD now have follow-ups of greater than one year, indicating a stable and durable response to treatment with CTX001. All patients with TDT and SCD experienced rapid and sustained increase in their total hemoglobin and fetal hemoglobin levels through last follow-up."
https://www.streetinsider.com/dr/news.php?id=17000083&gfv=1
The new crispr/cas9 treatment shows early flashes of what it could be in the future , a medical revolution.
CAMBRIDGE, Mass., Dec. 05, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced preclinical data and successful development of a large-scale manufacturing process for EDIT-301, a potentially best-in-class, one-time, durable, autologous cell therapy medicine to treat sickle cell disease and beta-thalassemia. EDIT-301 is the first experimental medicine in development generated using CRISPR/Cas12a gene editing. The Company reported these data today at the 62nd Annual Meeting and Exposition of the American Society of Hematology (ASH) being held virtually.
The data showed that high levels of editing in CD34+ cells from normal donors and sickle cell patients were achieved with CRISPR/Cas12a at the HBG1 and HBG2 promoters, leading to robust fetal hemoglobin (HbF) induction in their erythroid progeny in a pan-cellular fashion. Red blood cells derived from edited sickle cell patient CD34+ cells showed remarkable correction of sickle cell disease phenotypes, including a reduction in sickling and improved rheological properties when deoxygenated.
In addition, the Companyβs large-scale manufacturing process was shown to be consistent and robust. When infused into immunodeficient mice, edited CD34+ cells from normal donors manufactured at large-scale led to long term multi-lineage hematopoietic reconstitution that was comparable to unedited control cells. The engraftment was stable and highly polyclonal with high levels of editing detected throughout the course of the study.
βThese findings are very encouraging and further support our novel approach to developing and manufacturing EDIT-301 as a best-in-class and durable medicine for the potential treatment of sickle cell disease and beta-thalassemia,β said Charles Albright, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine. βIf these preclinical results translate to the clinic, we believe our editing approach may yield a safer and more effective medicine, addressing a significant unmet need for a transformative, durable treatment with the potential to transform the lives of people living with sickle cell disease and beta-thalassemia. β
Editas Medicine continues to prepare for a Phase 1/2 clinical trial evaluating EDIT-301 for the treatment of sickle cell disease. The Company has completed preclinical toxicology studies, identified a lead principal investigator, and engaged a contract research organization (CRO). Clinical tri
... keep reading on reddit β‘What is a good diet for me with this blood disorder?
Hey guys & gals, Would anyone be able to give me any tips about caring for someone with the above brand of sickle cell? She gets ongoing pain and acute painful crises sometimes that leave her unable to walk for short periods, but in general she's able to get up and go around for some of the day.
hello community, is there any parents with a children with sickle cell thalassemia? me (dad) caries the sickle cell trait and mom carries the thalassemia trait if we understood correct from the hospital few years back.
currently have 2 kids one 3 year old who has sickle cell thalassemia disease and one 1 year old who is normal. My son who has the disease takes medication every day, few ml of penicillin and 1 pill of folic acid and so far he's been on that since 6 months old till now and per doctors this will continue until 5 years old. I would consider him absolutely normal hes hyper, super smart even the doctors stay shocks with his conversations at 3 years of age, loves sports and riding his bicycle , he has a huge huge huge pain resistance since small, nothing seems to hurt him he walks it off and continues like nothing no matter how hard the fall is.
has far has crisis the only pain he suffers from would be leg pain during bed time here and there sometimes 3 times weekly or sometimes nothing at all for weeks. My way of treating this from the beginning has been with really potent (MG) CBD 'muscle freeze lotion' (3 different brands depending on stock) by massaging it to his crisis area and after 2 minutes he normally lets me know heβs s good to go and falls back asleep.
anyone in this community dealing with something similar and if you are what has worked for you during crisis episode or does it get worse depending on age?
Interviews for people (or their caregivers) with Sickle Cell, Prostate Cancer, or Thalassemia
Screener
Details:
60-minute Interview
From home
Eligibility criteria:
Adults who have been diagnosed with an eligible condition
NOTE: Only patients with verified diagnoses will be considered
Care partners of adults with an eligible condition
Eligible conditions are listed below.
Prostate cancer
Sickle-cell anemia
Thalassemia
Receive:
$110
https://gigs.savvy.coop/scpct/?r=602651
I have sickle cell trait and thalassemia type B but apparently I also lack vitamin D in the blood.
People often ask if I am high. I didnβt know what to tell them. Do these blood π©Έ disorders make people sluggish?
I am battling two blood disorders. How do I cope? I like reddit a lot because it is filled with people with my health condition. I struggle to find the energy I need to complete simple tasks.i recently bought a product recommended by a reddit user called ChlorOxygen. It helps boost red blood cells. I love it a lot. Please let me know other solutions I can take.
Sickle cell anemia and thalassemia are similar disorders. Both are caused by genetic mutations that result in malformed hemoglobin. In both cases a single copy of the gene will do little harm and confer a slight advantage against malaria, but two copies will result in serious disease.
I'm wondering if a child with one copy of the sickle cell gene and one copy of the thalassemia gene would also be at risk of serious disease. In other words, is it possible for two different but similar recessive genes to behave as if they were paired with copies of themselves?
Can anyone reference any source or explain differentiating sickle cell+thalassemia on electrophoresis?
The best interpretation I came across was from Medscape (http://emedicine.medscape.com/article/2085637-overview#a2).
So ultimately, how does one differentiate sickle cell trait vs. sickle alpha thalassemia? What about sickle beta0 thalassemia vs. sickle cell anemia (HbSS)?
Any input would be appreciated!
For the curious, parents who's newborns are diagnosed, and people who think it's "contagious." Ask away...
It totally isn't contagious btw...
Interviews for people (or their caregivers) with Sickle Cell, Prostate Cancer, or Thalassemia
Screener
Details:
60-minute Interview
From home
Eligibility criteria:
Adults who have been diagnosed with an eligible condition
NOTE: Only patients with verified diagnoses will be considered
Care partners of adults with an eligible condition
Eligible conditions are listed below.
Prostate cancer
Sickle-cell anemia
Thalassemia
Receive:
$110
https://gigs.savvy.coop/scpct/?r=602651
I have sickle cell trait and thalassemia type B but apparently I also lack vitamin D in the blood.
People often ask if I am high. I didnβt know what to tell them. Does these blood π©Έ disorders make people sluggish?
I am battling two blood disorders. How do I cope? I like reddit a lot because it is filled with people with my health condition. I struggle to find the energy I need to complete simple tasks.i recently bought a product recommended by a reddit user called ChlorOxygen. It helps boost red blood cells. I love it a lot. Please let me know other solutions I can take.
