Gene Therapy: Your Questions Answered

NORD’s RareEDU™ released this video, Gene Therapy: Your Questions Answered, in order to address a vital topic to today's rare disease community. The goal of this video is to address the questions, hopes and concerns that patients and caregivers, across many different diseases, have about gene therapy. Since more than 80% of rare diseases are believed to be genetic, this video will serve as a helpful resource for the rare disease community.

[@kathrynshaw192]

We need a stronger set of federal laws, safeguards and system of advocates to protect these NORD patients. Sometimes the state systems are inadequate, especially when the economy goes down, these patients are the first to lose access to their doctors, medications, homes, family and even their lives. The medical-research community has come so far in the last 50 years. It's a shame to see lives destroyed because of the greed and shortsighted view of a few.

[@adeelmehmood8093]

Praying for gene editing and gene therapy for polycystics kidney disease soon ameen

[@AmericanoSpidey]

Finally, someone knows how to explain the difference between gene therapy and gene editing

[@adeelmehmood8093]

True

[@ellios5734]

Thank you so much for such good explanation of gene therapy and gene editing

[@alisonrockettfrase7105]

Outstanding production NORD! well done

[@sharramoller7664]

So glad you exist! Haven't watched any videos, but will start now. When I tell a doctor that I have HTLV and should see an allergist, it's like I haven't said anything. I'm on my third doctor now trying to get a referral to an allergist. That, needing a referral, is not right. Hope someone changes that dumb and damaging law. Someone else said that if I am on SS I don't need a referral. Doesn't seem to be true even if it is true.

[@westfield90]

Scientist were so giddy about stem cells a decade ago but that went nowhere. I hope this shows more promise

[@Sylvia3456789w]

Praying for a cure for MTM🙏

[@fcludde6420]

not me

[@sharramoller7664]

Is there any gene therapy information related to HTLV I or II? Is it a logical possibility? What effects does it have on the patient while it is "working"? I also have more likely treatable conditions: narcolepsy, essential tremors, Hep B, hypothyroid, and ? Oh yeah, this memory problem :)

[@cindypalombo6111]

My son needs this

[@christiemint]

For gene therapy is it possible to do it with LCT gene? and if so where does the gene come from before being injected into the patient

[@troopstroops3021]

Is it possible for baldness?

[@tudormiller887]

I have Tyrosinemia Type 2.

[@clarriceestafia2080]

Should gene therapy be limited to medical concerns only (e.g., curing diseases) or could it be used as well for aesthetic purposes to enhance basic human traits such as height, intelligence, or athletic ability? Why do you say so?

[@hoangnamnguyen3779]

4 years passed, how can I access to an up-to-date progress of current approval ? Thank you

[@rameshjangra2178]

Nice vidio 😊☺️

[@anilkumarsasthramannil9130]

Gene terapy is possible for scoliosis

[@krzysiekskoczen1137]

I hope to live through gene therapy for my disease called congenital protein deficiency, I don't pray for it because I'm an atheist I ask everyone: keep your fingers crossed for this

[@amysesio3974]

Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?

[@ey8002]

Enough talk we want practical treatment

[@user-qv8ih7qi9o]

Gene therapy is possible for NBIA

[@zakirullakhan9000]

My two son die sma 1 and one son is Carrier i want mor baby no sma Deasise it posibal what for cure for sma diseases

[@sarleen9916]

Is this possible and cure heamophili A genetic disease lifetime cure options we will make choice is this gene therapy it is possible.. Heamophilia a gene therapy treatment yet 3rd stage on research going on the research team announced.. If avail this treatment can anyone tell me i suffer more to heamophilia life alive in india so we get new life begin start with we ready...

[@audreynicoletti7329]

Turn that loud music off

[@rahulpaul2976]

Is it possible to change skin color by gene therapy?

[@simplyexplained875]

Yeah, but why?

[@ellios5734]

Oh C'mon every skin colour is beautiful

[@umadev6077]

I hope generally Gene therapy is for those souls undergoing suffering between life and death ,hope it is covered by insurance companies and it should reach and available to the poorest deprived desperate children and victims hope the concerned authorities department of state and Central govt of our country and also other countries do the needful for treating genetic disorders at affordable amount reimbursement facilities and free for the poor with honest genuine dedicated declaration Vaazhga valamudan Uma s

[@umadev6077]

Is it possible for treating multiple neurofibromatosis type 1 and type 11 patients undergoing great pains and suffering both physically mentally and emotionally draining all the energies being avoided and insulted wounded teased mocked by the society, what will they do for their living and earning.kindly do something for treatment domicile follow-up expenses prevention measures remedies and solutions in future no children should be Born mentally retardant social sigma births leprosy multiple neurofibromatosis type 1 and type 11 blind deaf physically handicaped or suffer from life threatening diseases like cancer etc etc

[@sherlinefenelon818]

my son have x-ald now i am searching hel for him

[@indervirhundal7672]

Can it manipulate to create a normal brain from autistic one

[@toufiqkhan8783]

uses of gene therapy