Prime Editing Unlocks a Universal Strategy for Restoring Lost Proteins
November 22, 2025
Background
Epidemiology
Anatomy
Pathophysiology
Etiology
Genetics
Prognostic Factors
Clinical History
Physical Examination
Age group
Associated comorbidity
Associated activity
Acuity of presentation
Differential Diagnoses
Laboratory Studies
Imaging Studies
Procedures
Histologic Findings
Staging
Treatment Paradigm
by Stage
by Modality
Chemotherapy
Radiation Therapy
Surgical Interventions
Hormone Therapy
Immunotherapy
Hyperthermia
Photodynamic Therapy
Stem Cell Transplant
Targeted Therapy
Palliative Care
Medication
5
mg
Orally 
daily
w/wo tadalafil 20 mg PO qDay and after 4 weeks the dose of ambrisentan or tadalafil can be increased as PRN
Extended-release tablets:
Initial dose: 0.25 mg orally every 12 hours or 0.125 mg every 8 hours
Maintenance dose: Increase 0.125 mg 3 times a day every 3 to 4 days or increase 0.25 or 0.5 mg twice a day
Inhalation:
Initial dose: 4 times a day, three breaths (18 mcg) each treatment session; if not tolerated, reduce to 1 or 2 breaths; increase to 3 breaths as tolerated
Maintenance dose: Once the intended dose of 9 breathes (54 mcg) per treatment session is attained, increase by an additional three breaths at about 1-to-2-week intervals as tolerated
Maximum dose: 4 times daily treatment sessions of 9 breaths (54 mcg)
Injectable
Initial dose: Continuous subcutaneous or intravenous infusion of 1.25 ng/kg/min; if this dosage is intolerable, lower it to 0.625 ng/kg/min
Maintenance dose: During the first four weeks, increase the infusion rate by 1.25 ng/kg/min each week. Then, for the remaining four weeks, increase the rate by 2.5 ng/kg/min each week
Initial
:
Solution
Orally inhaled
inhale 2.5mcg then 5mcg in subsequent dose,6-9 times every day when necessary
Maintenance:2.5-5mcg/dose. Do not exceed 45mcg/day
Indicated for pulmonary arterial hypertension (PAH) :
10 mg once daily, taken orally with or without food.
Note:
Do not take more or less of the medication than directed, and do not stop taking it without first consulting your healthcare provider.
30
mg
Tablets
Orally 
every 12 hrs
can be increased to 120-240 mg/day
10
mg
Tablet
Orally 
every day
; may be increased up to 10-40mg and it is effective after 2 weeks
Do not exceed 80mg a day
40 mg orally once daily
May initiate with 20 mg daily and later increase to 40 mg 4 weeks later
For PAH, only Revatio is indicated, to delay the clinical worsening and improve clinical worsening
20 mg orally 3 times daily
If required, titrate the dose up to 80 mg 3 times daily
10 mg intravenous bolus 3 times daily
10 mg intravenous dose is predicted bioequivalent to 10 mg oral dose
Oral and intravenous doses should not exceed the recommended regimen
In the case of severe hepatic impairment like cirrhosis, keep an initial dose of 25 mg (for Viagra)
In the case of severe renal impairment, keep an initial dose of 25 mg (for Viagra)
:
For PAH, only Revatio is indicated, to delay the clinical worsening and improve clinical worsening
20 mg orally 3 times daily
If required, titrate the dose up to 80 mg 3 times daily
10 mg intravenous bolus 3 times daily
10 mg intravenous dose is predicted bioequivalent to 10 mg oral dose
Oral and intravenous doses should not exceed the recommended regimen
Orally
200 mcg orally twice a day initially
may Increase dosage by 200 mcg twice a day, generally weekly, to maximal tolerable dose; should not exceed more than 1600 mcg twice a day.
Reduce dosage if untolerable.
orally to Intravenous conversion
for patients temporarily not able to administer orally
Current dosage 1600 mg orally twice a day: Give 1800 mg intravenously twice a day
Current dosage 1400 mg orally twice a day: Give 1575 mg intravenously twice a day
Current dosage 1200 mg orally twice a day: Give 1350 mg intravenously twice a day
Current dosage 1000 mg orally twice a day: Give 1125 mg intravenously twice a day
Current dosage 800 mg orally twice a day: Give 900 mg intravenously twice a day
Current dosage 600 mg orally twice a day: Give 675 mg intravenously twice a day
Current dosage 400 mg orally twice a day: Give 450 mg intravenously twice a day
Current dosage 200 mg orally twice a day: Give 225 mg intravenously twice a day
A patch/pump drug device that offers a 2-day, disposable single-use Subcutaneous dosage for pulmonary arterial hypertension is pending FDA approval.
Administer 10mg/40mg tablet orally once a day.
Note: When the individual doses of tadalafil 40 mg and macitentan 10 mg once daily have stabilized, only then should the combined therapy be started.
100 mg is given orally once a day
<18 years: Safety and efficacy not established
Injectable (> 16 years)
Initial dose: 1.25 ng/kg/min subcutaneous/Intravenous infusion
Do not increase more than 1.25 ng/kg/min every week for four weeks
Oral tablet
Initial dose: 0.25mg orally, twice a day. Increase up to 0.5mg twice daily every 3-4 days
Maximum dose: 3.4mg twice a day
No dose adjustment is required in the case of renal or hepatic impairment:
Revatio is indicated for children of 1-17 years to treat pulmonary arterial hypertension For <20 kg: 10 mg orally 3 times daily For 20-45 kg: 20 mg orally 3 times daily For ≥45 kg: 20 mg orally 3 times daily In pediatrics, maximum dose cannot be identified
tadalafil is indicated for pulmonary arterial hypertension (PAH)
It improves the ability to exercise; studies establishing the efficacy included majorly the patients with NYHA Functional Class II – III symptoms. And the etiologies of heritable PAH (61%) are associated with PAH connective tissue diseases (23%)
Revatio is only indicated for PAH to delay clinical worsening and improve exercise ability
20 mg orally 3 times daily
If required, titrate the dose up to 80 mg orally 3 times daily based on tolerance and symptoms
10 mg intravenous bolus 3 times daily
10 mg intravenous dose is predicted
bioequivalent to 10 mg oral dose
Oral and intravenous doses should not exceed the recommended regimen
Future Trends
References

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